Ex Vivo Hsc Gene Therapy In Clinical Trials Flashcards
1
Q
Diseases involving Ex vivo HSC gene therapy in clinical trials
A
AD-SCID & BETA-THALASSEMIA
2
Q
AD-SCID & BETA-THALASSEMIA (rationale and target cells)
A
Lymphoid reconstitution; erythroid reconstitution
3
Q
AD-SCID & BETA-THALASSEMIA (gene vector)
A
Gamma-RV (MLV/SFFV LTR) expressing ADA;
SIN LV (large LCR expressing betaglobin and cHS4 insulator)
4
Q
AD-SCID & BETA-THALASSEMIA (safety)
A
Long-term safety demonstrated; clonal dominance possibly triggered by vector insertion in HMGA2 gene
5
Q
AD-SCID & BETA-THALASSEMIA (efficacy)
A
Long-term cure comparable to HCT;
Transfusion independence
