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Gene therapy is defined as a set of strategies that modify the expression of an individual’s genes or that correct abnormal genes. Each strategy involves.

Published byRebecca Hodge Modified over 6 years ago

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Presentation on theme: "Gene therapy is defined as a set of strategies that modify the expression of an individual’s genes or that correct abnormal genes. Each strategy involves."— Presentation transcript:

1 Gene therapy is defined as a set of strategies that modify the expression of an individual’s genes or that correct abnormal genes. Each strategy involves the administration of a specific DNA (or RNA). Cell therapy is defined as the administration of live whole cells or maturation of a specific cell population in a patient for the treatment of a disease. – Bone Marrow Transplantation

2 Bone Marrow Transplantation for DBA
Can cure the disease Widely available Graft failure Only ¼ siblings is likely to be a good Donor - Need to know mutation Good unrelated donors are rare

3 Gene Therapy for Immune Disorders
MACROPHAGE STEM CELLS ERYTHROCYTES PLATELETS T CELLS B CELLS BASOPHILS EOSINOPHILS NEUTROPHILS COMMITTED PROGENITOR CELL

4 Gene Therapy for ADA Deficiency
STEM CELLS ADA ADA T CELLS B CELLS COMMITTED PROGENITOR CELL ERYTHROCYTES BASOPHILS PLATELETS EOSINOPHILS NEUTROPHILS MACROPHAGE 4

5 Gene Therapy for ADA Deficiency
MACROPHAGE STEM CELLS ERYTHROCYTES PLATELETS T CELLS B CELLS BASOPHILS EOSINOPHILS NEUTROPHILS COMMITTED PROGENITOR CELL Virus containing a normal ADA gene ADA 5

6 Gene Therapy for ADA Deficiency

7 Gene Therapy for ADA Deficiency
Collect Marrow Isolate CD34+ Cells SCF MGDF FL Combine Aliquots Backup Marrow MND-ADA GCsapM-ADA Transduce x 3 Days Busulfan 75 mg/m2 ADA- Discontinue PEG-ADA Reinfuse ADA?

8 Gene Therapy for X-SCID:
A Cautionary Tale

9 Gene Therapy for Hemoglobin Disorders

10 Gene Therapy for Hemoglobin Disorders

11 Why isn’t there Gene Therapy for More Diseases?
Funding for vector development: $$$$$ FDA approval for Investigational New Drug: Time Institutional approval: Time RAC approval: Time Patient recruitment: Fast Vector production: $$$$

12 Gene Therapy for DBA Vector development is simple
Procedures in place for related diseases Regulatory process is getting easier Need to develop vectors First trials will be on adults not children Risks associated with transplant and insertion

13 Genome Editing: The Next Big Thing
Mice Fish

14 Gene Editing Targeted to a single place
in the genome Works well in animal models Delivery is complex – CAS9, guide and cargo Gene disruption is more common than homologous recombination Off target effects

15 When will we see Gene Editing in the Clinic?
Improve homologous recombination frequency Efficient delivery without insertion Off target effects? Success in animal models Funding for trial development FDA approval for IND…..

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