Dr. Robert G. Pergolizzi - Class Site
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Background

Dr. Robert Pergolizzi was recently recruited from the Department of Genetic Medicine at Weill Cornell in New York City, where he performed gene therapy and stem cell research for the past six years. He has an extensive publication record, with over seventy publications in top-tier scientific journals. He was involved in formal teaching of classical genetics and genetic medicine to medical students, and was awarded the prestigious “Excellence in Teaching” Award by Cornell University. Dr. Pergolizzi was the Director of a National Gene Vector Lab at Cornell, one of five in the country funded by the National Heart, Lung and Blood Institute of the National Institutes of Health. His research at Cornell focused on curing genetic diseases by using genes as medicine and development of novel methods to deliver genes that are too large or too toxic for conventional gene transfer vectors. He developed a technique, called “Segmental Trans-splicing” which permits the delivery of such genes in pieces for intracellular reassembly and expression. This research has been published in Nature Medicine, Molecular Therapy and Cancer Research, and has received much international attention. This strategy is being used by other researchers, in major NIH-funded program projects on cystic fibrosis and muscular dystrophy. He also directed several projects involving embryonic stem cells, to develop these powerful cells as an alternative treatment modality for human disease.

Prior to his work at Weill Cornell, Dr. Pergolizzi was Director of Molecular Genetics and Gene Therapy for the North Shore-Long island Jewish Health System for fourteen years, where his lab developed tests for genetic diseases, including a patented test which could identify carriers of the gene for the fragile-X syndrome for the first time. He directed a highly successful NIH-funded program for minority students, aimed at encouraging them to pursue careers in science and medicine. He designed and built a GMP Gene Therapy Lab and fully implemented FDA regulations and validation, thus permitting the products of the lab to be used in human clinical trials.

He received the PhD degree from the Department of Biochemistry and Molecular Biology at Columbia University, and then did a post-doctoral fellowship in Human Genetics and Development at the same institution. During that period, he had the opportunity to teach molecular biology to gifted high school students in the Science Honors Program sponsored by the National Science Foundation, an extremely positive experience that formed the basis for his interest in teaching at the Bergen Academies today.

Dr. Pergolizzi enjoys running, all types of music and he loves good food and spending time with his three sons, one of whom attended the Bergen Academies. With his background of developing the tools and basic principles for using stem cells and genes as medicines, he is looking forward to being a resource for the students at the Academies, teaching and helping them develop successful biological research projects.

 

Personal Statement

My interests have evolved from chemistry to biochemistry and molecular biology and ultimately to genetic medicine. My graduate training provided a thorough familiarity with nucleic acid and protein chemistry, with particular emphasis on modified bases and amino acids. My early research focused on the biosynthesis of modified nucleotides, their relevance to disease, especially cancer, and the effects of environmental carcinogens on the coding properties of DNA and RNA. I developed strategies for DNA sequencing (when it was still avant garde, and used those strategies to clone and identify the first regulatory mutations in a human gene (beta globin/beta thalassemia). My work at Columbia was funded by NIH, the American Cancer Society and the Brookdale Foundation.

My attention turned to the development of non-radioactive DNA-based assays for infectious diseases, and developed and patented several methods for signal amplification. Over four years I received four of the first NIH SBIR grants awarded, for a total of over 2.5 million dollars. A diagnostic test that I developed (for Herpes Simplex viruses 1 and 2 infection in gynecologic specimens) was approved for human use by the FDA, and I was obliged to learn and implement the required Good Manufacturing Procedures (GMP) to produce and market the test. This knowledge became useful again in the development of the GMP Viral Vector Lab at North Shore –Long Island Jewish (LIJ) Health System.

As Director of Molecular Genetics for the North Shore-LIJ Health System, my lab focused on genetic testing, developing rapid screens for oncogene mutations, and a method for isolating large viral genomes from human cells. My lab developed a method for screening the human genome for mutations, which was applied to chromosome 22 in an effort to localize the causative factor in meningioma as part of the Human Genome Initiative. I developed a PCR-based test which could detect the carrier state for the fragile-X syndrome for the first time, which allowed the identification of families at risk for having affected children. This test was patented and published in the Lancet. To educate my colleagues on the emerging field of genetic medicine, I developed a curriculum and offered a well-attended 12 session course on molecular biology and the use of genes in medicine. I also directed a highly successful NIH-funded program for minority students, to encourage them to pursue careers in science and medicine. This program continued for nine years

During my tenure at North Shore I was de facto Chief Operating Officer for the research enterprise, handling all aspects of space allocation, equipment purchase and maintenance, budgets, and interfacing with all departments of the Health System to facilitate operations. I was a member of the hospital Institutional Review Board, Radioisotope Use and Ethics committees, and chairman of the Institutional Biosafety Committee. Through the affiliation of North Shore with Cornell Medical College and later at the New York University Medical College, I rose to the academic rank of Associate Professor in Clinical Pathology. When an interest arose in developing Gene Therapy research, I was named Director of Gene Therapy and given responsibility for the planning and development a research program in this area, and to design and build a GMP Viral Vector Lab and an ancillary major lab expansion project. All aspects of development of the lab were under my direct control, including fund-raising, design, staffing and implementation of FDA regulations and validation, thus permitting the products of the lab to be used in human clinical trials. The 5.5 million dollar projects were completed, on time and under budget. The GMP Vector Lab continues to produce clinical grade vectors for numerous projects, many of which are in current clinical trials, including projects related to breast and ovarian cancer, wound healing and angiogenesis. The GMP Vector Lab that I designed and built has been a model for several other labs, including the GMP Vector lab at Weill Cornell. In fact, visits by the Cornell faculty to the GMP lab at North Shore ultimately resulted in my recruitment to the Department of Genetic Medicine at Weill Cornell.

Since my arrival at Cornell, my attention has been focused on developing gene therapy as a viable paradigm for the treatment of human disease. I am currently the director of a large group embracing several diverse studies, including a hemophilia study, the goal of which is to deliver the very large von Willebrand factor(VWF) gene to cells and tissues, and ultimately to ameliorate the symptoms of von Willebrand disease in a human clinical trial. Using a mouse VWF knockout model, we have developed a novel strategy for gene delivery. Since the gene is too large to fit into an appropriate vector, we developed a technique, called “Segmental Trans-splicing” which permits the delivery of large or toxic genes in pieces for intracellular reassembly and expression. This research has been published in Nature Medicine, Molecular Therapy and Cancer Research, and has received much attention. This strategy is also being used in other group projects, including a major NIH-funded program project on cystic fibrosis. In addition, I direct several projects involving murine and human embryonic stem cells, using them as vehicles for gene transfer for the correction of several diseases, including alpha-1-antitrypsin deficiency and hereditary tyrosinemia. The stem cell research is part of a new initiative involving Weill Cornell, Rockefeller University and Memorial Sloan-Kettering Cancer Center, to develop these powerful cells as an alternative treatment modality for human disease.

At Weill Cornell, I am involved in formal teaching of classical genetics and genetic medicine to medical students. I have been awarded the prestigious “Excellence in Teaching” Award by Cornell University. I genuinely enjoy teaching and I look forward to the opportunity to devote myself to it. I feel that I have a genuine ability to motivate young minds to slice through the superficial complexity of these scientific disciplines to the underlying unifying principles and beauty that lies beneath the surface. I would also like to write a science text for young people

My background in a variety of scientific disciplines has given me a different perspective than most of my colleagues in the hospital setting, and I have often been able to add diversity to projects by means of an alternate approach. My philosophy has been and remains one of service to the institution and to society, and I have been pleased and honored to assist many scientists and physicians in their efforts. My experience has provided a forum for me to apply this philosophy to practical matters. Importantly, I have also demonstrated talent as a highly effective fund raiser. As can be clearly seen from my publication record, the evolution of my career has allowed me to move from defining disease at the genetic level to the development of diagnostic tools to identify individuals and families who are afflicted with or carriers for genetic diseases, and, during the last several years, to developing the tools and basic principles for using genes as medicine. I firmly believe that the power of this approach will usher in a new age of medicinal chemistry, in which drug design will be assisted by directed evolution within living cells.

In summary, I feel I have a skill set which include scientific, managerial, administrative, regulatory and operational, with essentially unique perspective and experience in GMP manufacture and facilities design of medicines of the future. I have experience in program and curriculum development, and teaching which I find especially rewarding, both as a lecturer and as a mentor. Working with the students at the Academy is an exciting new chapter in my career. I developed a deep sense of respect for the Academies when one of my sons (Alex) attended as a student. Working with these exceptional young people, and mentoring them through their emerging interest in research, is an opportunity and a responsibility that I have thought about for many years.

CURRICULUM VITAE

Robert G. Pergolizzi, PhD

Bergen County Academies
Department of Biology
200 Hackensack Avenue, Room 244
Hackensack, NJ 07601


Phone: (201) 343-6000 ext. 3306
e-mail: robper@bergen.org

EDUCATION:

Columbia University, Graduate School of Arts and Sciences, N.Y.
1979 Ph.D. Biochemistry
1978 M.Phil. Biochemistry
1976 M.A. Biochemistry

Hofstra University, Hempstead, N.Y.
1974 M.A. Biology
1971 B.S. Chemistry

PROFESSIONAL POSITIONS HELD:

Columbia University, N.Y., N.Y.
Postdoctoral Fellow, Department of Human Genetics and Development 1/80-5/82

Enzo Biochem, Inc., N.Y., N.Y.
Director, Recombinant DNA Laboratory 5/82-6/86
Director of Manufacturing (GMP compliant) 8/84-6/86

North Shore-Long Island Jewish Health System, Manhasset, N.Y.
Director, Molecular Genetics, Department of Research 8/86-6/98
Director, Instrument Facility, Biomedical Research Center 8/86-1/01
Director, Molecular Biology, Department of Pathology 7/87-1/01
Director, DNA Diagnostic Lab, Department of Pediatrics 3/89-1/01
Co-Director, Transgenic Animal Facility 7/89-1/01
Director, Core Services, North Shore University Hospital 1/90-1/01
Director, Gene Therapy and GMP Vector Lab 8/95-1/01

Weill Medical College of Cornell University, NY, N.Y.
Senior Research Associate 2/01-Present
Director, GMP Viral Vector Laboratory 6/05-Present

Angion Biomedica Corporation, Great Neck, New York
Member, Scientific Advisory Board
Consultant in Gene Therapy and GMP Vector Production 10/00-Present

Enzo Biochem, Inc., N.Y., N.Y.
Consultant in Gene Therapy and GMP Vector Production 10/98-Present

Bergen County Academies, Hackensack, NJ
Research Specialist, 9/06-Present

ACADEMIC APPOINTMENTS

Weill Medical College of Cornell University, New York, N.Y.
Assistant Professor of Pathology 7/87-10/93
Associate Professor of Clinical Pathology 10/93-7/95

New York University Medical College, New York, N.Y.
Associate Professor of Clinical Pathology 7/95-1/01

Weill Medical College of Cornell University, New York, N.Y
Senior Research Associate in Genetic Medicine 2/01-4/06
Associate Professor in Genetic Medicine 4/06-9/06

MEMBERSHIPS:
American Society of Human Genetics
International Society for Stem Cell Research
American Society of Gene Therapy
New York Academy of Sciences
Harvey Society
American Association for Cancer Research
American Society of Hematology
Association of Clinical Scientists
NCI Cancer Center Review Committee

HONORS AND AWARDS:
Weill Cornell Medical College “Excellence in Teaching" Award
Columbia University Fellowship
Brookdale Foundation Award
Waldemar Foundation Award
Who’s Who in America
Who’s Who in the World
Who’s Who in Science and Medicine
Who's Who in American Education

BIBLIOGRAPHY

JOURNAL ARTICLES:

1. Pergolizzi R.G., Teichberg S., Lifshitz F., Wapnir R. (1977) Interaction Between Dietary Carbohydrates and Intestinal Disaccharides in Experimental Diarrhea, American Journal of Clinical Nutrition, 30; 482-489.

2. Lifshitz F., Wapnir R., Wehman H., Diaz-Bensussen S., Pergolizzi R.G. (1978) Effects of Small Intestinal Colonization by Fecal and Colonic Bacteria on Intestinal Function in Rats, Journal of Nutrition, 108; 1913-1923.

3. Teichberg S., Lifshitz F., Pergolizzi R.G., Wapnir R. (1978) Response of Rat Intestine to a Hyperosmotic Feeding, Pediatric Research, 12; 720-725.

4. Pergolizzi R.G., Engelhardt D.L., Grunberger D. (1978) Formation of Phenylalanine Transfer RNA Lacking the Wye Base in Vero Cells during Methionine Starvation, Journal of Biological Chemistry, 253; 6341-6343.

5. Singer B., Pergolizzi R.G., Grunberger D. (1979) Synthesis and Coding Properties of Dinucleoside Diphosphates Containing Alkyl Pyrimidines which are formed by the action of Carcinogens on Nucleic Acids, Nucleic Acids Research, 6; 1709-19.

6. Pergolizzi R.G., Engelhardt D.L., Grunberger D. (1979) Incorporation of Lysine into Y Base of Phenylalanine-tRNA in Vero Cells, Nucleic Acids Research, 6; 1709-19.

7. Grunberger D., Pergolizzi R.G., Jones R.E. (1980) Translation of Globin Messenger RNA Modified by Benzo(a)pyrene 7,8-Dihydrodiol 9,10-Oxide in a Wheat Germ Cell-Free System, Journal of Biological Chemistry, 255; 390-394.

8. Pergolizzi R.G., Grunberger D. (1980) The Effect of Exogenous Nutrients on the Biosynthesis of the Y Base in tRNAphe from Ehrlich Ascites Carcinoma Cells, Cancer Letters, 8; 329-334.

9. Pergolizzi R.G., Spritz R.A., Goosens M., Spence S., Kan Y.W., Bank A. (1981) Two Cloned Beta-Thalassemia Genes are Associated with Amber Mutations at Codon 39, Nucleic Acids Research, 9; 706-72.

10. Spence S., Pergolizzi R.G., Donovan-Peluso M., Kosche K., Dobkin C.S., Bank A. (1982) Five Nucleotide Changes in the Large Intervening Sequence of a Beta Globin Gene in a Beta+ Thalassemia Patient, Nucleic Acids Research, 10; 1283-1294.

11. Bank A, Baird M, Driscoll MC, Pergolizzi RG, Spence SE, The gene defects in the beta-thalassemias. Birth Defects Orig Artic Ser 1982;18:45-50

12. Dobkin C.S., Pergolizzi R.G., Bahre P., Bank A. (1983) Abnormal Splice in a Mutant Beta-Globin Gene not at the Site of the Mutation, Proc Natl Acad Sci, 80; 1184-1188.

13. Grunberger D, Pergolizzi RG, Kuchino Y, Mushinski JF, Nishimura S, (1983) Alterations in post-transcriptional modification of the Y base in phenylalanine tRNA from tumor cells. Recent Results Cancer Res;84:133-45

14. Molden D.P., Nakamura R.M., Suzuki H., Greer S., Brakel C., Pergolizzi R.G. (1985) Comparison of Radiolabeled DNA Probe With a Non-isotopic Probe for Assay of Serum Hepatitis B Virus DNA, Clin Physiol Biochem, 3(4); 174-83.

15. Sun T., Susin M., Desner M., Pergolizzi R.G., Cuomo J. (1990) The Clonal Origin of Two Cell Populations in Richter’s Syndrome. J. Hum.Pathol. 21; 722-728.

16. Pergolizzi R.G., Brown W.T., Gross A., Goonewardena P., Dobkin C. (1991) Molecular Characterization of a DNA Probe, U6.2, Located Close to the Fragile X Locus. Am. J. Medical. Genet. 38; 380-383.

17. Pergolizzi R.G., Rottach C., Kreis W. (1993) Analysis of the K-Ras Mutational Status of Prostrate Carcinoma Cell Lines by Oligonucleotide Hybridization of DNA Amplified by Polymerase Chain Reaction, Cancer Investigation, 11; 25-32.

18. Pergolizzi, R.G., Erster, S.H., Goonewardena, P., and Brown, W.T. (1992) Detection of the Full Fragile X Mutation by Polymerase Chain Reaction, Lancet, 339; 271-272.

19. Erster, Susan H., Brown W. T., Goonewardena, P., and Dobkin, C.S., Jenkins, E.C., Pergolizzi, R.G. (1992) PCR Analysis of Fragile X Mutations, Human Genetics, 90; 55-61.

20. Hingorami, R., Choi, I., Akolkar, P., Gulwani-Akolkar, B., Pergolizzi, R.G., Silver J., Gregersen, P. (1993), Clonal predominance of T cell receptors within the CD8+ CD45RO+ subset in normal human subjects. J Immunol;151:5762-9

21. Koduru, P.R.K., Goh, J.C., Pergolizzi, R.G., Lichtman, S.M. and Broome, J.D. (1993) Molecular Characterization of a Variant Ph Translocation t(9;22;11)(q34;q11;q13) in Chronic Myelogenous Leukemia (CML) Reveals the Translocation of the 3’-part of BCR Gene to the Chromosome Band 11q13. Oncogene, 8; 3239-3247.

22. Snow, K. Doud, L.K., Hagerman, R., Pergolizzi, R.G., Erster, S.H., and Thibodeau, S.N. (1993) Analysis of a CGG Sequence at the FMR-1 Locus in Fragile X Families and the General Population. Am. J. Hum. Genet., 53; 1217-1228.

23. Akolkar, P., Gulwani-Alkolkar, B., Pergolizzi, R., Bigler, R.D. and Silver, J. (1993) The Influence of HLA Genes on TCR V-Segment Frequencies and Expression Levels in Peripheral Blood Lymphocytes. J Immunol;150:2761-73

24.. Pergolizzi RG, Erster SH, (1994) Analysis of chromosome 22 loci in Meningioma. Alterations in the leukemia inhibitory factor (LIF) locus., Mol Chem Neuropathol;21:189-217

25. Akolkar, P.N., Chirmule, N., Gulwani-Alkolkar, V.S., Pahwa, S., B., Kalyanaraman, Pergolizzi, R.G., Macphail, S. and Silver, J. (1995) V beta-specific Activation of T Cells by the HIV Glycoprotein. Scandinavian J. Immunol., 41; 487-498.

26. Goodman, S. Sawada, T., Barbosa, J., Cole, B., Pergolizzi, R.G., Silver, J., Mellins, E., Chang, M.D. (1995) Mutational Analysis of Two DR Alpha Residues Involved in Dimers of HLA-DR Molecules. J. Immunol., 155; 1210-1217.

27. Akolkar, P.N., Gulwani-Alkolkar, Chirmule, N., V.S., Pahwa, S., B., Kalyanaraman, Pergolizzi, R.G., Macphail, S. and Silver, J. The HIV Glycoprotein gp160 has Superantigen-like Properties (1995) Clinical Immunology and Immunopathology, 76; 255-265.

28. Monteiro, J., Hingorani R, Choi IH, Silver J, Pergolizzi R., Gregersen, P.K. (1995) Oligoclonality in the Human CD8+ T Cell Repertoire in Normal Subjects and Monozygotic Twins: Implications for Studies of Infectious and Autoimmune Diseases. Mol. Med., 1; 614-24.

29. Monteiro, J., Hingorani R., Choi I.H., Pergolizzi R., Silver J., Gregersen, P.K. (1995) Variability in CD8+ T-Cell Oligoclonality Patterns in Monozygotic Twins. Ann. N.Y. Acad. Sci, 756; 96-8.

30. Akolkar P.N., Gulwani-Akolkar B., Chirmule N., Pahwa S., Kalyanaraman V.S., Pergolizzi R., Macphail S, Silver, J. (1995) V Beta-Specific Activation of T-Cells by the HIV Glycoprotein gp160. Ann. N.Y. Acad. Sci., 756; 447-9.

31. Hingorani, R., Monteiro, J., Pergolizzi, R., Furie, R., Chartash, E., Gregersen P.K. (1995) CDR3 Length Restriction of T-Cell Receptor Beta Chains in CD8+ T-Cells of Rheumatoid Arthritis Patients. Ann N.Y. Acad. Sci., 756; 179-82.

32. Monteiro J, Hingorani R, Pergolizzi R, Apatoff B, Gregersen PK, (1995) Clonal dominance of CD8+ T-cell in multiple sclerosis. Ann N Y Acad Sci;756:310-2

33. Shepp, D. H., Match, M.E., Ashraf, A.B., Lipson, S.M., Millan, C., and Pergolizzi, R.G. (1996) Cytomegalovirus Glycoprotein B Groups Associated with Retinitis in AIDS. J. Infectious Diseases, 174; 184-7.

34. Hingorani R, Monteiro J, Furie R, Chartash E, Navarrete C, Pergolizzi R, Gregersen PK, (1996) Oligoclonality of V beta 3 TCR chains in the CD8+ T cell population of rheumatoid arthritis patients. J Immunol;156:852-8

35. Sawada, T., Pergolizzi, R.G., Ito, K., Silver, J., Atkin, B., Cole, B., and Chang, M. D. (1995) Replacement of the DR Alpha Chain with the E Alpha Chain Enhances Presentation of Mycplasma Arthritis Superantigen by the Human Class II DR Molecule. Infection and Immunity, 63; 3367-3372.

36. Goodwin, L.O., Leeds, N.B., Hurley, I., Mandel, F.S., Pergolizzi, R.G., Benoff, S. (1997) Isolation and Characterization of the Primary Structure of Testis-Specific L-Type Calcium Channel: Implications for Contraception Mol Hum Reprod;3:255-68

37. Shepp DH, Match ME, Ashraf AB, Lipson SM, Millan C, Pergolizzi R, (1996) Cytomegalovirus glycoprotein B groups associated with retinitis in AIDS. J Infect Dis;174:184-7

38. Shepp, D.H., Ashraf, A., Tang, I.T., Match, M. E., Millan, C., Pergolizzi, R. (1996) Reverse Transcriptase Genotype and Antiretroviral Susceptibility of Human Immunodeficiency Virus Isolates from Patients with Advanced Disease Treated with Didanosine: Correlation with Virologic Response and Survival. J. Med. Virol., 49; 303-10.

39. Gulwani-Akolkar B., Akolkar, P.N., Minassian, A., Pergolizzi, R., McKinley, M., Mullin, G., Fisher, S., Silver, J. (1996) Selective Expansion of Specific T Cell Receptors in the Inflamed Colon of Crohn’s Disease. J. Clin Invest, 98; 1344-54.

40. Ito, K., Fetten, J., Khalili, H., Hajdu, S., Busch, E., Pergolizzi, R., Vinciguerra, V., Chang, M.D. (1997) Oligoclonality of CD8+ T Cells in Breast Cancer Patients. Mol Med, 3; 836-51.

41. Donson, D., Borrero, H., Rutman, M., Pergolizzi, R., Malhado, N., Macphail, S. (1997) Gene Transfer Directly Demonstrates a Role for TCR V Alpha Elements in Superantigen Recognition. J Immunol, 158; 5229-36.

42. Hingorani, R., Monteiro, J., Furie, R., Chartash, E., Navarette, C., Pergolizzi, R., Gregersen, P.K. (1996) Oligoclonality of V Beta 3 TCR Chains in the CD8+ T-Cell Population of Rheumatoid Arthritis Patients. J Immunol, 156; 852-8.

43. Tolbert, W.R., Merchant, B., Taylor, J.A., and Pergolizzi, R.G., Designing an initial gene therapy manufacturing facility, BioPharm, 9, 32-40 (1996).

44. Deshpande, R., Khalili, H., Pergolizzi, R.G., Michael, S.D., Chang, M.D. (1997) Estradiol Down-Regulates LPS-Induced Cytokine Production and NFkB Activation in Murine Macrophages. Am J Reprod Immunol, 38; 46-54.

45. Mostardini M, Appierto V, Pergolizzi R, Zucchi I, Mumm S, DeBellis G, Milanesi L, Rogozin IB, Biunno I, (1997) Identification of a U7snRNA homologue mapping to the human Xq27.1 region, between the DXS1232 and DXS119 loci. Gene;187:221-4

46. Goodwin, L.O., Leeds, N.B., Hurley, I., Cooper, G.W., Pergolizzi, R.G., Benoff, s. (1998) Alternative Splicing of Exons in the Alpha 1 Subunit of the Rat Testis L-Type Voltage-Dependent Calcium Channel Generates Germ Line-Specific Dihydropyridine Binding Sites. Mol Hum Reprod, 4; 215-26.

47. Shepp, D.H., Match, M.E., Lipson, S.M., Pergolizzi, R.G. (1998) A Fifth Human Cytomegalovirus Glycoprotein B Genotype. Res Virol, 149; 109-14.

48. Lipson, S.M., Match, M.E., Shepp, D.H., Lotlikar, M.S. Teichberg, S., Pergolizzi, R. (1998) Identification of an Exogenous Retrovirus (Foamy Virus Type 1) in Rhesus Monkey Kidney Cell Culture: Significance to Viral Diagnostics. J Clin Virol, 11; 149-53.

49. Mason, J.M., Grande, D.A., Barcia, M., Grant, R., Pergolizzi, R.G., Breitbart, A.S. (1998) Expression of Human Bone Morphogenic Protein 7 in Primary Periosteal Cells: Potential Utility in Gene Therapy for Osteochondral Repair. Gene Ther, 5; 109-114.

50. Fitness J, Dixit N, Webster D, Torresani T, Pergolizzi R, Speiser PW, Day DJ, (1999) Genotyping of CYP21, linked chromosome 6p markers, and a sex-specific gene in neonatal screening for congenital adrenal hyperplasia. J Clin Endocrinol Metab; 84:960-6

51. Mason JM, Guzowski DE, Goodwin LO, Porti D, Cronin KC, Teichberg S, Pergolizzi RG. (1999) Human serum-resistant retroviral vector particles from galactosyl (alpha1-3) galactosyl containing nonprimate cell lines. Gene Ther;6(8):1397-405.

52. Breitbart AS, Mason JM, Urmacher C, Barcia M, Grant RT, Pergolizzi RG, Grande DA. (1999) Gene-enhanced tissue engineering: applications for wound healing using cultured dermal fibroblasts transduced retrovirally with the PDGF-B gene. Ann Plast Surg.43(6):632-9.

53 Mason, J.M., Breitbart, A.S., Barcia, M., Porti, D., Pergolizzi, R.G., and Grande, D.A. (2000)
Cartilage and Bone Regeneration Using Gene-Enhanced Tissue Engineering. Clinical Orthopediatics and Related Research; 379, S171-178

54. Goodwin LO, Karabinus DS, Pergolizzi RG. (2000) Presence of N-cadherin transcripts in mature spermatozoa. Mol Hum Reprod. 6:487-97.

55. Goodwin LO, Leeds NB, Guzowski D, Hurley IR, Pergolizzi RG, Benoff S. (1999) Identification of structural elements of the testis-specific voltage dependent calcium channel that potentially regulate its biophysical properties. Mol Hum Reprod. 5(4):311-22.

56. Sondhi, D,. Hackett, N R,. Apblett, R L,. Kaminsky, S M, Pergolizzi, R G, and. Crystal, RG (2001) Feasibility of Gene Therapy for Late Neuronal Ceroid Lipofuscinosis Arch Neurol.,58 (11): 1793-1798

57. Frieri M, Pergolizzi R, Millan C (2000). Dominguez PJ. Cytokine, chemokine, and nitric oxide (NO) release in stimulated small airway epithelial cells (SAEC) treated with ß2 -agonist enantiomers of albuterol. J Allergy Clin Immunology, 105: S23

58. S. M. Lipson, L. Svenssen, L. Goodwin, D. Porti, S. Danzi and R. Pergolizzi (2001) Evaluation of two current generation enzyme immunoassays and an improved isolation-based assay for the rapid detection and isolation of rotavirus from stool . Journal of Clinical Virology, 21; 17-27

59. Zhang F, Hackett NR, Lam G, Cheng J, Pergolizzi R, Luo L, Shmelkov SV, Edelberg J, Crystal RG, Rafii S (2003) Green fluorescent protein selectively induces HSP70-mediated up-regulation of COX-2 expression in endothelial cells Blood;102:2115-21

60. Pergolizzi RG, Ropper AE, Dragos R, Reid AC, Nakayama K, Tan Y, Ehteshami JR, Coleman SH, Silver RB, Hackett NR, Menez A, Crystal RG. (2003) In vivo trans-splicing of 5' and 3' segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer. Mol Ther; 8: 999-1008

61. Zhang F, Cheng J, Hackett NR, Lam G, Shido K, Pergolizzi R, Jin DK, Crystal RG, Rafii S. Adenovirus E4 gene promotes selective endothelial cell survival and angiogenesis via activation of the VE-cadherin/Akt signaling pathway. J Biol Chem 2004; 279:11760-11766.

62. Pergolizzi RG, Crystal RG. (2004)Genetic medicine at the RNA level: Modifications of the genetic repertoire for therapeutic purposes by pre-mRNA trans-splicing. Comptes Rendus Biologies; 327: 695-709

63. Frank Y, Pergolizzi RG, Perilla MJ. Dopamine (2004) D4 receptor gene and attention deficit hyperactivity disorder. Pediatr Neurol. 31(5):345-8.

64. Tahara M, Pergolizzi RG, Kobayashi H, Krause A, Crystal RG. (2004) Trans-splicing correction of CD40 ligand deficiency resulting in naturally regulated correction of a murine model of hyper IgM X-linked immunodeficiency. Nat Med; 10:834-841.

65. Nakayama, K., Pergolizzi, R.G. and Crystal, R.G (2005) Gene Transfer–Mediated Pre-mRNA Segmental Trans-splicing As a Strategy to Deliver Intracellular Toxins for Cancer Therapy. Cancer Res; 65(1): 254-63

66. Pergolizzi RG, Dragos R, Ropper AE, Menez A, Crystal RG. (2005) Protective immunity against alpha-cobratoxin following a single administration of a genetic vaccine encoding a non-toxic cobratoxin variant. Hum Gene Ther.16(3):292-8.

67. Benoff S, Goodwin LO, Millan C, Hurley IR, Pergolizzi RG, Marmar JL. (2005) Deletions in L-type calcium channel alpha1 subunit testicular transcripts correlate with testicular cadmium and apoptosis in infertile men with varicoceles. Fertil Steril. 83(3):622-34.

68. Benoff S, Goodwin LO, Millan C, Hurley IR, Pergolizzi RG and Marmar JL (2006) Deletions in L-type calcium channel INCLUDEPICTURE "http://humrep.oxfordjournals.org/math/alpha.gif" \* MERGEFORMATINET 1 subunit testicular transcripts correlate with testicular cadmium and apoptosis in infertile men with varicoceles. Fertil Steril 83, in press

70. Zhang, F., Cheng, J., Lam, G., Boyer, JL, Pergolizzi, R., Karajannis, MA, Hackett, NR, and Rafii, S (2006) Adenovirus E4)RF1 Regulation of PI3K and FGF-2 Modulates the Angiogenic Phenotype of Endothelial Cells. (Manuscript submitted to Blood)

BOOKS:
Pergolizzi, R.G. (Editor), Genetic Engineering/Biotechnology Sourcebook (1981)
McGraw-Hill., N.Y.

BOOK CHAPTERS:

1. Bank A, Burns AL, Baird M, Pergolizzi RG. Globin Gene Pathology Clues to Gene Function and Hemoglobin Switching. Stamatoyannopoulos G and Nienhius AW. (Ed.) Organization and Expression of Globin Genes. Vii+339p. Alan R. Liss, Inc., New York, NY, USA (1981) 69-278.

2. Bank A, Burns AL, Baird M, Pergolizzi RG. Globin Switching. Organ Expression Globin Genes. Proc. Conf. Hemoglobin Switching, 2nd, 269-278 (1981).

3. Bank A, Baird M, Driscoll MC, Pergolizzi RG, Spene SE. The Gene Defects in the Beta Thalassemias. Cao, A, Carcassi, U, Rowley, PT (Ed.) National Foundation March of Dimes Birth Defects Original Article Series, Vol. 18, No. 7, Thalassemia: Recent Advances in Detection and Treatment; International Symposium, Cagliari, Sardinia, Italy, June 7-11, 1981. Alan R. Liss, Inc.: New York, NY, USA. (1982) P45-50.

4. Grunberger D, Pergolizi RG, Kuchino Y, Mushinski JF, Nishimura 5. Alteration in Post Transcriptional Modification of the Y Base in Phenylalanine Transfer RNA of Tumor Cells. Workshop on Modified Nucleotides and Cancer, Preiburg Im Breisgau, West Germany, Sept. 28 - Oct. 2, 1981. Cancer Res Clin Oncol. 103(3) (1982) 316-317.

5. Bank A, Spence SE, Pergolizzi RG, Driscoll MC, Baird M. Gene Defects in the Beta Thalassemias. Univ. Chicago Sickle Cell Cent. Hemoglobin Symp. 3, 41-9 (1983).

6. Grunberger D, Pergolizzi RG, Kuchino Y, Mushinski JF, Nishimura 5. Alterations in Post-Transcriptional Modifications of the Y Base in Phenylalanine tRNA from Tumor Cells. Recent Results Cancer Res. 84, 133-45 (1983).

7. Pergolizi, R.G. and Erster, S.H. Analysis of Chromosome 22 Loci in Meningioma: Alterations in the Leukemia Inhibitory Factor (LIF) Locus. (1994) Proceedings of the Chicago Institute for Neurobiology and Neurosciences in Molecular and Chemical Neuropathology, 21: 189-2178.

8. Brown, W.T., Goonewardena, P., Gross, A.C., Pergolizzi, R.G., Dobkin, C., Jenkins, E.C. Molecular Markers of Fragile X: Recent Research Developments. Proceedings of the Albany Birth Defects Symposium XX

9. Pergolizzi RG, Crystal RG. Cystic fibrosis transmembrane conductance regulator gene and protein. Encyclopedia of Respiratory Medicine Editors: GJ Laurent and SD Shapiro 2006 Elsevier Ltd

10. Pergolizzi, RG and Crystal RG. Gene Therapy in The Mouse in Biomedical Research, 2nd Ed. (2006) Elsevier Science Press, Ltd. Philadelphia, PA, (in press)

ABSTRACTS (partial listing):

1. Lifshitz F, Pergolizzi RG, Wapnir RA. Intestinal Disaccharides in Experimental Osmotic Diarrhea. Pediatr Res, 8(40), 38, 1974.

2. Lifshitz F, Wapnir RA, Wehman HJ, Hawkins RL, Pergolizzi RG. Enteric Microbial Effects on Jejunal Carbohydrate Transport Disaccharidase Activity Bile Salts and Ultrastructure. Gastroenterology, 66(4), 73, 1974.

3. Teichberg S, Lifshitz F, Pergolizzi RG, Wapnir RA. Response of Rat Intestine to an Acute Hyper Osmotic Feeding. Pediatr Res, 9, 309, 1975.

4. La-Sala MA, Lirshitz F, Pergolizzi RG, Silverberg M, Wapnir RA. Magnesium Metabolism in Patients with Chronic Inflammatory Disease of the Bowel. Am J Clin Nutr, 28(4), 42, 1975.

5. Pergolizzi RG, Lifshitz F, Teichberg S, Wapnir RA. Experimental Osmotic Diarrhea Carbohydrate Intake Effects on Intestinal Disaccharidases. Fed Proc, 34(3), 90, 1975.

6. Lifshitz F, Wapnir RA, Pergolizzi RG, Teichberg S. Hypoxia Effects on Carbohydrate Transport. Pediatr Res, 10(4), 35, 1976.

7. Lifshitz F, Pergolizzi RG, Lipkin A, Teichberg S, Wapnir RA. Alterations in Intestinal Transport and Sodium Plus Potassium Ion Activated ATPase Hypoxia. Fed Proc, 35(3), 46, 1976.

8. Lifshitz F, Wapnir RA, Wehman H, Pergolizzi RG, Teichberg S. Intestinal Function Alterations Induced by Enteric Bacterial Proliferation. Proc Xth Int’l Cong Nutr, Tokaroike, Sakyo-ku, Kyoto, Japan, Aug. 1975, 220. #4239, 1976.

9. Lifshitz F, Pergolizzi RG, Lipkin A, Teichberg S, Wapnir RA. Alterations in Intestinal Transport and (Na+K+) -ATPase in Hypoxia. Fed Proc, 35:464, 1976.

10. Pergolizzi RG, Engelhardt DL, Grunberger D. Regulation of Abundance of a Phenylalanine Transfer RNA Species Lacking the Hyper Modified Y Base Methionine Starvation in a Monkey Kidney Cell Line. Fed Proc, 37(6), 176, 1978.

11. Pollice M, Yang H, Silver S, Brakel C, Thalenfeld B, Pergolizzi RG. Intracellular Bacterial Infections Non-Radioactive Detection of Chlamydia Trachomatis by In-situ DNA Hybridization. 24th Annual Meeting of the Aamerican Society for Cell Biology, Kansas City, MO, USA, Nov. 12-16, 1984. J Cell Biol, 99 (Part 2). 249A, 1984.

12. Thalenfeld BE, Pergolizzi RG, Solanki M, Marshak A, Engelhardt D. In-situ Detection of Viral Infections Using Nonradioactive DNA Hybridization. 84th Annual Meeting of the American Society for Microbiology, St Louis, MO, USA, Mar. 4-9, 1984. Annual Meet Am Soc Microbiol, 84(10). Abstract C69.

13. Stavrianopoulos J, Pergolizzi RG, Engelhardt D, Brakel C. A Novel Method for Labeling and Detection of DNA Using Radioactive Metals. 69th Annual Meeting of the Federation of American Societies for Experimental Biology, Anaheim, Calif., USA, Apr. 21-26, 1985. Fed Proc, 44(5), 1622 (1985).

14. Bank A, Spence SE, Pergolizzi RG, Donovan-Peluso M, Kosche KA, Dobkin CS. Gene Defects in the Beta Thalassemias. 11th Annual Cetus-UCLA Symposium on Gene Regulation, Squaw Valley, Calif., USA, Mar. 28 - Apr. 3, 1982. J Cell Biochem, Suppl. 10(6), 297, 1987.

15. Pergolizzi RG, Kaplan MH. (1988) Detection and Isolation of Human Herpesvirus, Type 6, Using Pulsed-Field Gel Electrophoresis. Clin Res, 36, 801.

16. Pergolizzi RG, Brown NA, Kaplan MH. (1988) Pulsed-Field Gel Electrophoresis in the Analysis and Isolation of Human Herpesvirus (Type 6) Genomes. Blood, 72, (Suppl.) 168a.

17. Goonewardena, P., Pletcher, BA, Pergolizzi, RG, and Brown, WT (1989) Use of PCR with Y-Specific Probes for Rapid Sex Determination (Abstract 743), American Journal of Human Genetics Supplement 45: A190.

18. Brown WT, Goonewardena P, Gross A, Pergolizzi RG, Ferrando C, Dobkin C, Jenkins E, Dahl N, Pettersson U. (1989). Characterization and Use of a New Tightly-Linked DNA Probe (U6.2) in the Fragile-X Syndrome. (Abstract 514, American Journal of Human Genetics, 45: A190.

19. Pergolizzi RG, Rottach C, Kreis W. Analysis of the K-Ras Mutational Status of Prostate Carcinoma Cell Lines by Oligonucleotide Hybridization of DNA Amplified by Polymerae Chain Reaction. Proceedings of the American Association for Cancer Research, 31:225 (1990).

20. Pergolizzi, R.G., Erster, S. H., Nikolopolous, S., Waber, P., Farmer, P., and Broome, J. Molecular Analysis of the Meningioma Locus, Proceedings of the American Association for Cancer Research, 31: 38, (1990).

21. Shah, J., Sun, T., Pieciak, W., Pergolizzi, R.G., Tananbaul, B., and Lane, D.J. RNA Probes for the Diagnosis of Pneumocystis Carinii. (Proceedings of the VIIth International Congress of Parisitology, 1990).

22. Schuster, M.W., Allen, S.L., Schorschinksy, N., Pergolizzi, R.G., and Koduru, P. Failure of the Polymerase Chain Reaction to Demonstrate BCR Gene Rearrangement in a Patient with Ph Negative CML and Isochrome 17. (Proceedings of the American Society of Clinical Oncology, 1990).

23. Sawada, T., Pergolizzi, R.G., Atkin B., Silver, J., Cole, B., and Chang, M.D. (1994) MAM has a Higher Affinity of the Ea Chain than the Dra chain Arthritis and Rheumatism 37:166.

24. Goodman, S., Sawada, T., Pergolizzi, R.G., Silver, J., Amaya, M., Mellins, E., and Chang, M.D. Mutational Analysis of Two Dra2 Alleles involved in DR-DR Dimerization. (1994) Arthritis and Rheumatism 37:291.

25. Nakayama K,.Hackett NR, Pergolizzi RG, Jiang Q, Crystal RG. Segmental gene delivery and in vitro assembly of functional expression cassettes by a novel trans-splicing method. Mol Ther 2002; 5: S138

26. Tahara M, Hackett NR, Pergolizzi RG, Crystal RG. In vitro trans-splicing-mediated CD40-ligand gene therapy for X-linked immunodeficiency. Mol Ther 2002;5: S394

27. Pergolizzi RG, Hackett NR, Lee JS, Lam M, Patel MN, Song YS, Zheng C, Baum BJ, Singh RN Crystal RG. Delivery and expression of Von Willebrand factor cDNAs via hybrid adenovirus-retrovirus vectors in vitro and in vivo. Mol Ther 2002; 5: S84

28. Pergolizzi RG, Hackett NR,. Lee JS, Lam M, Patel MN, Song YS, van’t Hof W, Zheng C, Baum BJ, Singh RN, Crystal RG. Adenovirus vectors containing retrovirus LTRs mediate different patterns of in vivo gene expression than routine adenovirus vectors. Mol Ther 2002; 5: S57-S58

29. Zhang F, Hackett NR, Pergolizzi R, Cheng J, Crystal RG, Rafii S. Adenovirus E4 promotes selective endothelial cell survival via the Akt signaling pathway. Mol Ther 2003;7:S57

30. Zhang F, Hackett NR, Pergolizzi R, Cheng J, Crystal RG, Rafii S. Adenovirus E4 promotes selective endothelial cell survival via the Akt signaling pathway. Mol Ther 2003;7:S57;

31. Zhang F, Hackett NR, Lam G, Shmelkov S, Pergolizzi R, Luo L, Cheng J, Edelberg J, Crystal RG, Rafii S. Green fluorescent protein selectively induces HSP70-mediated upregulation of COX-2 expression in endothelial cells. Mol Ther 2003;7:S243-S244;

32. Zhang F, Hackett NR, Pergolizzi R, Cheng J, Crystal RG, Rafii S. Adenovirus E4 promotes selective endothelial cell survival via the Akt signaling pathway. Mol Ther 2003;7:S57;

33. Zhang F, Hackett NR, Lam G, Shmelkov S, Pergolizzi R, Luo L, Cheng J, Edelberg J, Crystal RG, Rafii S. Green fluorescent protein selectively induces HSP70-mediated upregulation of COX-2 expression in endothelial cells. Mol Ther 2003;7:S243-S244;

34. Zhang F, Hackett NR, Lam G, Shmelkov S, Pergolizzi R, Luo L, Cheng J, Edelberg J, Crystal RG, Rafii S. Green fluorescent protein selectively induces HSP70-mediated upregulation of COX-2 expression in endothelial cells. Mol Ther 2003;7:S243

35. Pergolizzi RG, Ropper AE, Dragos R, Reid AC, Nakayama K, Silver RB, Hackett NR, Menez A, Crystal RG. In vivo trans-splicing of 5' and 3' segments of pre-mRNA directed by corresponding dna sequences delivered by gene transfer. Mol Ther 2003;7:S21

36. Nakayama K, Pergolizzi, RG, Tan Y, Hackett NR, Crystal RG. Gene therapy-based intracellular cytotoxicity using the shigatoxin 1a1 gene. Mol Ther 2003;7:S421

37. Tahara M, Hackett NR, Pergolizzi RG, Crystal RG. Trans-splicing-mediated CD40-ligand gene therapy correction of a murine model of X-linked immunodeficiency. Mol Ther 2003;7:S154

38 Pergolizzi RG, Dragos R, Ropper A, Menez A, Crystal RG. Development of a genetic vaccine conferring protective immunity against α-cobratoxin following a single administration of an adenovirus vector encoding a modified, non-toxic cobratoxin variant. Mol Ther 2004;9:S143

39. Nakayama K, Pergolizzi RG, Crystal RG. Intracellular shigatoxin 1A1 delivered by adenovirus-mediated segmental trans-splicing causes apototic cell death in vitro and in vivo. Mol Ther 2004;9:S253

40. Pergolizzi RG, Dragos R, Nakayama K, Wagner D, Crystal RG. Overcoming the size limitations of gene transfer vectors: Using segmental trans-splicing to deliver the coding sequence of Von Willebrand factor. Mol Ther 2004;9:S61

41. Tahara M, Pergolizzi RG, Crystal RG .Trans-splicing correction of CD40 ligand deficiency resulting in naturally regulated correction of a murine model of hyper IgM X-linked immunodeficiency. Mol Ther 2004;9:S271

42.Boyer JL, Howard J, Hackett NR, Pergolizzi RG, Wilson JM, Crystal RG. Persistent expression of single chain antibodies mediated by AAV5 and AAVrh.10 vectors. Mol Ther 2006; (in press)