Clinical Trials of Genetic Therapy With Antisense DNA and DNA Vectors: Wickstrom, Eric: 9780824700850: Book: : eCampus.com

Thomas Jefferson Univ., Philadelphia, PA. Presents illustrative examples of disease in which gene- based interventions are plausible, as well as case studies of current research using both synthetic oligonucleotides and biological vectors. For pharmacologists, biochemists, pathologists, and medical and graduate students. DNLM: Gene Therapy.

Foreword: Genetic Therapy in the 21st Century

iii

(2)

O. Michael Colvin

Preface: From Theory to Practice in Thirty Years

(8)

Eric Wickstrom

Contributors

xiii

1. A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics

(38)

James W. Hawkins

2. Preclinical Development of Antisense Oligonucleotide Therapeutics for Cancer: Regulatory Aspects

(14)

Chang-Ho Ahn

Joseph J. DeGeorge

3. Commercial Scale Manufacturing of Oligonucleotides Under Good Manufacturing Practices

(36)

Jose E. Gonzalez

Richard G. Einig

Patricia Puma

Timothy P. Noonan

Paul E. Kennedy

Bruce G. Sturgeon

Bing H. Wang

Jin-yan Tang

4. The Regulatory Process and Gene Therapy

(12)

Suzanne L. Epstein

5. Production of Clinical Lots of Gene Therapy Vectors Using Good Manufacturing Practice: Experience in a University Setting

101

(10)

Alan R. Davis

Colleen Baker

6. Gene Therapy Clinical Trials for Adenosine Deaminase Deficiency/Severe Combined Immunodeficiency

111

(22)

Erlinda M. Gordon

W. French Anderson

7. Development of an Oligodeoxynucleotide Pharmaceutical for the Treatment of Human Leukemia

133

(22)

Alan M. Gewirtz

Deborah Lee Sokol

8. Clinical Trials with Anti-p53 DNA, OL(1)p53, in Patients with Acute Myelogenous Leukemia and Myelodysplastic Syndrome

155

(14)

Patrick L. Iversen

9. Human Bcl-2 Antisense Therapy for Lymphomas

169

(22)

Finbarr E. Cotter

Andrew Webb

Paul Clarke

David Cunningham

10. Retroviral Gene Transfer in Autologous Bone Marrow and Stem Cell Transplantation

191

(28)

Rafat Abonour

Kenneth Cornetta

11. Adenoviral Gene Transfer of the Herpes Virus Thymidine Kinase Gene for Treating Gliomas

219

(20)

Jane B. Alavi

Jason G. Smith

Stephen L. Eck

12. Distribution and Toxicity of Retroviral Vectors After Intracavitary Delivery in Mouse and Man

239

(18)

Patrice S. Obermiller

Anne M. Pilaro

Carlos L. Arteaga

Jeffrey T. Holt

13. Clinical and Immunologic Responses to Gene Transfer of an Allogeneic Major Histocompatibility Complex Antigen

257

(22)

Alison T. Stopeck

Evan M. Hersh

14. Defective Tumor Suppressor Gene Replacement and Oncogene Inactivation for the Treatment of Cancer

279

(16)

Jack Roth

15. The Molecular Basis of Bladder Cancer and Prospects for Gene Therapy Using Hammerhead Ribozymes

295

(16)

Eric J. Small

Mohammed Kashani-Sabet

David Y. Bouffard

Kevin J. Scanlon

16. In Situ Gene Insertion for Immunotherapy Using Vaccinia Virus Vectors

311

(20)

Edmund C. Lattime

Laurence C. Eisenlohr

Michael J. Mastrangelo

17. Antisense Oligonucleotide-Based Therapy for HIV-1 Infection from Laboratory to Clinical Trials

331

(22)

Sudhir Agrawal

18. Treatment of Retinitis Induced by Cytomegalovirus Using Intravitreal Fomivirsen (Isis 2922)

353

(10)

Stanley T. Crooke

19. Synthetic DNA-Based Compounds for the Prevention of Coronary Restenosis: Current Status and Future Challenges

363

(32)

Andrew Zalewski

Yi Shi

John D. Mannion

Fernando Roque

20. Prevention of Restenosis by Gene Targeting

395

(18)

Michael J. Mann

Heiko E. Von der Leyen

Victor J. Dzau

Index

413




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