Disclosure

Gene Therapy

Gene Replacement/Homologous Recombination

Gene Addition

Vectors for Gene Transfer

Slide 6

Adenoviruses

Adeno-associated Viruses

Retroviruses

Uses of Gene Therapy

Gene Therapy Versus Protein Therapy

Systems to Study Gene Transfer

Factor 8 and 9 Deficiencies

Ischemic Vascular Disease

Anti-Cancer Gene Therapy

Adding a Toxic Gene

Anti-Sense to Oncogenes

Tumor Suppressor Genes

Increase Anti-tumor Immune Responses

Cancer Gene Therapy

Slide 21

Slide 22

Slide 23

Critical Plasmids for Safe Retroviral Production

MDR Gene Therapy

Slide 26

Slide 27

Slide 28

Slide 29

Slide 30

MDR Transduction in Mice

Challenges of Human Gene Therapy

Slide 33

Autotransplantation

Peripheral Blood Stem Cells

Progenitor Assays

Transduction Protocol

Summary

Columbia MDR Phase1Clinical Trial

Requirements for HSC Gene Transfer

Murine Studies-Qin 1999

Indiana Trial- MDR Gene Therapy

Slide 43

Indiana Gene Therapy Trial

Slide 45

Indiana Trial: Summary

NOD-SCID Mouse Assay

NOD-SCID Mouse Engraftment

MDR-Transduced HSC in
NOD-SCID Mouse - MDR PCR

Summary: MDR-Transduced HSC in NOD-SCID Mouse

Amphotropic Retroviral Packaging Lines

VSV-G Envelope Packaging Lines

RD114 Envelope Packaging Lines

Stable RD114 Packaging Line (M. Ward)

Current Bank lab GT Goals-2003

Cure of Children with X-SCID

Leukemia  in  Children with X-SCID

Lentiviral Vectors

Slide 59

Lentiviral Plasmids

Slide 61

Successful b Thal Gene Therapy

Successful Sickle Gene Therapy

Sickle Mouse Models

Leboulch Globin Lentiviral Vector

Slide 66

Current Gene Therapy Experiments - 4/03