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 Bone Marrow Transplantation for DBA ID: 1033086
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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 DBACan cure the diseaseWidely availableGraft failureOnly ¼ siblings is likely to be a good Donor - Need to know mutationGood unrelated donors are rare
3. MACROPHAGESTEMCELLSERYTHROCYTESPLATELETST CELLSB CELLSBASOPHILSEOSINOPHILSNEUTROPHILSCOMMITTEDPROGENITOR CELLGene Therapy for Immune Disorders
4. ADAADAMACROPHAGESTEMCELLSERYTHROCYTESPLATELETST CELLSB CELLSBASOPHILSEOSINOPHILSNEUTROPHILSCOMMITTEDPROGENITOR CELLGene Therapy for ADA Deficiency
5. MACROPHAGESTEMCELLSERYTHROCYTESPLATELETST CELLSB CELLSBASOPHILSEOSINOPHILSNEUTROPHILSCOMMITTEDPROGENITOR CELLVirus containing a normal ADA geneADAGene Therapy for ADA Deficiency
6. Gene Therapy for ADA Deficiency
7. CollectMarrowIsolateCD34+CellsSCFMGDFFLCombineAliquotsBackup MarrowMND-ADAGCsapM-ADATransduce x 3 DaysBusulfan75 mg/m2ADA-DiscontinuePEG-ADAReinfuse ADA?Gene Therapy for ADA Deficiency
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: $$$$$FDA approval for Investigational New Drug: TimeInstitutional approval: TimeRAC approval: TimePatient recruitment: FastVector production: $$$$
12. Gene Therapy for DBAVector development is simpleProcedures in place for related diseasesRegulatory process is getting easierNeed to develop 18-20 vectorsFirst trials will be on adults notchildrenRisks associated with transplantand insertion
13. MiceFishGenome Editing: The Next Big Thing
14. Gene EditingTargeted to a single place in the genomeWorks well in animal modelsDelivery is complex – CAS9, guide and cargoGene disruption is more common thanhomologous recombinationOff target effects
15. When will we see Gene Editing in the Clinic?Improve homologous recombination frequencyEfficient delivery without insertionOff target effects?Success in animal modelsFunding for trial developmentFDA approval for IND…..