"Regulatory Perspectives on Cellular Therapeutics"
Raj Puri, M.D., Ph.D.
Acting Director, Division of Cellular and Gene Therapies
Chief, Laboratory of Molecular Tumor Biology

Office of Cellular, Tissue and Gene Therapies

FDA, Center for Biologics Evaluations and Research

Jakubzick C, Kunkel SL, Joshi BH, Puri RK, Hogaboam CM. Interleukin-13 Fusion Cytotoxin Arrests Schistosoma mansoni Egg-Induced Pulmonary Granuloma Formation in Mice.  Am J Pathol. 2002 Oct;161(4):1283-97.

Kawakami K, Kawakami M, Puri RK. Cytokine receptor as a sensitizer for targeted cancer therapy.Anticancer Drugs. 2002 Aug;13(7):693-9. 

Kawakami K, Kawakami M, Husain SR, Puri RK. Targeting interleukin-4 receptors for effective pancreatic cancertherapy.  Cancer Res. 2002 Jul 1;62(13):3575-80. 

Joshi BH, Kawakami K, Leland P, Puri RK. Heterogeneity in interleukin-13 receptor expression and subunit structure in squamous cell carcinoma of head and neck: differential sensitivity to chimeric fusion proteins comprised of interleukin-13 and a mutated form of pseudomonas exotoxin.
Clin Cancer Res. 2002 Jun;8(6):1948-56. 

Blease K, Schuh JM, Jakubzick C, Lukacs NW, Kunkel SL, Joshi BH, Puri RK, Kaplan MH, Hogaboam CM. Stat6-deficient mice develop airway hyperresponsiveness and peribronchial fibrosis during chronic fungal asthma.
Am J Pathol. 2002 Feb;160(2):481-90. 

Kawakami M, Kawakami K, Puri RK. Apoptotic pathways of cell death induced by an interleukin-13 receptor-targeted recombinant cytotoxin in head and neck cancer cells.Cancer Immunol Immunother. 2002 Feb;50(12):691-700. 

Joshi BH, Leland P, Silber J, Kreitman RJ, Pastan I, Berger M, Puri RK. IL-4 receptors on human medulloblastoma tumours serve as a sensitive target for a circular permuted IL-4-Pseudomonas exotoxin fusion protein.  Br J Cancer. 2002 Jan 21;86(2):285-91. 

Strome SE, Kawakami K, Alejandro D, Voss S, Kasperbauer JL, Salomao D, Chen L, Maki RA, Puri RK. Interleukin 4 receptor-directed cytotoxin therapy for human head and neck squamous cell carcinoma in animal models. Clin Cancer Res. 2002 Jan;8(1):281-6. 

Kawakami K, Kawakami M, Leland P, Puri RK. Internalization property of interleukin-4 receptor alpha chain increases cytotoxic effect of interleukin-4 receptor-targeted cytotoxin in cancer cells.
Clin Cancer Res. 2002 Jan;8(1):258-66. 

Kawakami K, Kawakami M, Snoy PJ, Husain SR, Puri RK. In vivo overexpression of IL-13 receptor alpha2 chain inhibits tumorigenicity of human breast and pancreatic tumors in immunodeficient mice.
J Exp Med. 2001 Dec 17;194(12):1743-54.

"New Families of Simian Viruses as Vectors for Gene Therapy"
James M. Wilson, M.D., Ph.D.
Professor of Medicine

Division of Medical Genetics
University of Pennsylvania School of Medicine

Gonzalez-Aseguinolaza G, Van Kaer L, Bergmann CC, Wilson JM, Schmieg J, Kronenberg M, Nakayama T, Taniguchi M, Koezuka Y, Tsuji M. Natural killer T cell ligand alpha-galactosylceramide enhances protective immunity induced by malaria vaccines. J Exp Med. 2002 Mar 4;195(5):617-24.

Xiang Z, Gao G, Reyes-Sandoval A, Cohen CJ, Li Y, Bergelson JM, Wilson JM, Ertl HC. Novel, chimpanzee serotype 68-based adenoviral vaccine carrier for induction of antibodies to a transgene product. J Virol. 2002 Mar;76(6):2667-75.

Wang X, Moser C, Louboutin JP, Lysenko ES, Weiner DJ, Weiser JN, Wilson JM. Toll-like receptor 4 mediates innate immune responses to Haemophilus influenzae infection in mouse lung. J Immunol. 2002 Jan 15;168(2):810-5.

Raper SE, Yudkoff M, Chirmule N, Gao GP, Nunes F, Haskal ZJ, Furth EE, Propert KJ, Robinson MB, Magosin S, Simoes H, Speicher L, Hughes J, Tazelaar J, Wivel NA, Wilson JM, Batshaw ML.  A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase
deficiency. Hum Gene Ther. 2002 Jan 1;13(1):163-75.

Schnell MA, Hardy C, Hawley M, Propert KJ, Wilson JM.  Effect of blood collection technique in mice on clinical pathology parameters. Hum Gene Ther. 2002 Jan 1;13(1):155-61.

Cohen CJ, Xiang ZQ, Gao GP, Ertl HC, Wilson JM, Bergelson JM.  Chimpanzee adenovirus CV-68 adapted as a gene delivery vector interacts with the coxsackievirus and adenovirus receptor. J Gen Virol. 2002 Jan;83(Pt 1):151-5.

Auricchio A, Kobinger G, Anand V, Hildinger M, O'Connor E, Maguire AM, Wilson JM, Bennett J. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model.  Hum Mol Genet. 2001 Dec 15;10(26):3075-81.

Zoltick PW, Wilson JM. Regulated gene expression in gene therapy.
Ann N Y Acad Sci. 2001 Dec;953:53-63. Review.

Farina SF, Gao GP, Xiang ZQ, Rux JJ, Burnett RM, Alvira MR, Marsh J, Ertl HC, Wilson JM. Replication-defective vector based on a chimpanzee adenovirus. J Virol. 2001 Dec;75(23):11603-13.

Odaka M, Sterman DH, Wiewrodt R, Zhang Y, Kiefer M, Amin KM, Gao GP, Wilson JM, Barsoum J, Kaiser LR, Albelda SM.  Eradication of intraperitoneal and distant tumor by adenovirus-mediated interferon-beta gene therapy is attributable to induction of systemic immunity. Cancer Res. 2001 Aug 15;61(16):6201-12.

Rusconi CP, Scardino E, Layzer J, Pitoc GA, Ortel TL, Monroe D, Sullenger BA. RNA aptamers as reversible antagonists of coagulation factor IXa. Nature. 2002 Sep 5;419(6902):90-4.

Sullenger BA, Gilboa E. Emerging clinical applications of RNA.

Nature. 2002 Jul 11;418(6894):252-8. Review.

Martell RE, Nevins JR, Sullenger BA. Optimizing aptamer activity for gene therapy applications using expression cassette SELEX. Mol Ther. 2002 Jul;6(1):30-4.

White R, Rusconi C, Scardino E, Wolberg A, Lawson J, Hoffman M, Sullenger B. Generation of species cross-reactive aptamers using "toggle" SELEX. Mol Ther. 2001 Dec;4(6):567-73.

Rusconi CP, Yeh A, Lyerly HK, Lawson JH, Sullenger BA.  Blocking the initiation of coagulation by RNA aptamers to factor VIIa. Thromb Haemost. 2000 Nov;84(5):841-8.

Sullenger BA. Series introduction: emerging clinical applications of nucleic acids

J Clin Invest. 2000 Oct;106(8):921-2. No abstract available.

White RR, Sullenger BA, Rusconi CP. Developing aptamers into therapeutics.

J Clin Invest. 2000 Oct;106(8):929-34. No abstract available.

Guo H, Karberg M, Long M, Jones JP 3rd, Sullenger B, Lambowitz AM. Group II introns designed to insert into therapeutically relevant DNA target sites in human cells.

Science. 2000 Jul 21;289(5478):452-7.

Watanabe T, Sullenger BA. Induction of wild-type p53 activity in human cancer cells by ribozymes that repair mutant p53 transcripts. Proc Natl Acad Sci U S A. 2000 Jul 18;97(15):8490-4.

Long MB, Sullenger BA. Evaluating group I intron catalytic efficiency in mammalian cells.

Mol Cell Biol. 1999 Oct;19(10):6479-87.
 

Director, Laboratory of Viral Onco-Therapeutics

Hermelin Brain Tumor Center, Henry Ford Hospital

Ahmed M, Lock M, Miller CG, Fraser NW. Regions of the herpes simplex virus type 1 latency-associated transcript that protect cells from apoptosis in vitro and protect neuronal cells in vivo. J Virol. 2002 Jan;76(2):717-29.

Miller CG, Krummenacher C, Eisenberg RJ, Cohen GH, Fraser NW. Development of a syngenic murine B16 cell line-derived melanoma susceptible to
destruction by neuroattenuated HSV-1. Mol Ther. 2001 Feb;3(2):160-8.

Poptani H, Duvvuri U, Miller CG, Mancuso A, Charagundla S, Fraser NW, Glickson JD, Leigh JS, Reddy R. T1rho imaging of murine brain tumors at 4 T.
Acad Radiol. 2001 Jan;8(1):42-7.

Miller CG, Fraser NW. Role of the immune response during neuro-attenuated herpes simplex virus-mediated tumor destruction in a murine intracranial melanoma model. Cancer Res. 2000 Oct 15;60(20):5714-22.

Kotwal GJ, Miller CG, Justus DE.  The inflammation modulatory protein (IMP) of cowpox virus drastically diminishes the tissue damage by down-regulating cellular infiltration resulting from complement activation.
Mol Cell Biochem. 1998 Aug;185(1-2):39-46.

Miller CG, Shchelkunov SN, Kotwal GJ. The cowpox virus-encoded homolog of the vaccinia virus complement control protein is an inflammation modulatory protein. Virology. 1997 Mar 3;229(1):126-33.

Miller CG, Cook DN, Kotwal GJ.  Two chemotactic factors, C5a and MIP-1alpha, dramatically alter the mortality from zymosan-induced multiple organ dysfunction syndrome (MODS): C5a contributes to MODS while MIP-1alpha has a protective role. Mol Immunol. 1996 Oct;33(14):1135-7.

Garner WL, Rodriguez JL, Miller CG, Till GO, Rees RS, Smith DJ, Remick DG.
Acute skin injury releases neutrophil chemoattractants.
Surgery. 1994 Jul;116(1):42-8.

Rodriguez JL, Miller CG, Garner WL, Till GO, Guerrero P, Moore NP, Corridore
M, Normolle DP, Smith DJ, Remick DG. Correlation of the local and systemic cytokine response with clinical outcome following thermal injury.
J Trauma. 1993 May;34(5):684-94; discussion 694-5.

Rodriguez JL, Miller CG, DeForge LE, Kelty L, Shanley CJ, Bartlett RH,
Remick DG. Local production of interleukin-8 is associated with nosocomial pneumonia. J Trauma. 1992 Jul;33(1):74-81; discussion 81-2.