Gene Therapy

Prepared by:

Joanne T. Imperial
MaEd-Teaching Science
 Different Routes of Gene Therapy
• Ex vivo gene therapy -
• Usually with blood cells (lymphocytes or blood
stem cells) for diseases affecting the hematopoietic
system

• In vivo gene therapy -

• Oncolytic adenoviruses for the treatment of cancer
• Adeno-associated vectors for the treatment of
Duchenne muscular dystrophy or hemophilia
• Non-viral for cancer
Gene Therapy in Blood Cells
Gene Therapy in Blood Cells

Therapeutic HIV
protection gene
Gene Therapy in Blood Cells
Gene Therapy in Blood Cells
Gene Therapy in Blood Cells

Therapeutic HIV
protection gene
Some targets for gene therapy
Some targets for gene therapy
Some targets for gene therapy
Some targets for gene therapy
Some targets for gene therapy
Next Generation COOH

Technology Zinc finger

Genome editing
 Zinc finger NH2

 TAL Effector TAL Effector

Nuclease
Nuclease
CRISPR/Cas9
 CRISPR/Cas9

megaTAL
 MegaTals
 HSC Modification Results in the Development of Infection Resistant
Immune Cell Populations and an Enhanced Immune Response

Development of Gene Modified,

Infection Resistant CD4+ T-cells

Cytolytic Activity
R5- tropic X4- tropic
Dampening Long-term
of IR protection

Maintenance
Resistance to B-Cell of SHIV- CD8+ T-Cell
Direct Infection function Specific CD4+ function
T-Cells

Peripheral Maintenance of
Tolerance Macrophage Lymphoid Tissue
Dual-tropic
Activation

Decreased Viremia Younan…Kiem Blood 2013

REFERRENCES:

HIV Cure Research Training Curriculum

Cell/Gene Therapy by:

Jeff Sheehy, the California Institute for Regenerative Medicine

(CIRM)

Jerome Zack, UCLA

Hans-Peter Kiem, The Fred Hutchinson Cancer Research Center

Jessica Handibode, AVAC

Acknowledgements