Table 1.
Clinical studies for gene and cell therapy of hemophilia A and B.
| Disease | Vector | Route of administration |
Results | Ref. |
|---|---|---|---|---|
| Hemophilia A | Transient transfection of hFVIII into autologous dermal fibroblasts |
Transplantation of fibroblasts into omentum |
Transient elevation of FVIII levels in three out of six patients |
[3] |
| Hemophilia A | Retroviral vector expressing B-domain deleted hFVIII |
Intravenous: peripheral vein |
Transient elevation of FVIII levels in nine out of 13 patients | [4] |
| Hemophilia A | Gutless adenoviral vector expressing full-length hFVIII |
Intravenous | Toxic reaction in one patient, resulting in discontinuation of treatment |
[5] |
| Hemophilia B | rAAV2 vector expressing hFIX | Intramuscular: skeletal muscle |
Effective gene transfer into muscle tissue. Therapeutic levels of FIX were not seen |
[29–31] |
| Hemophilia B | rAAV2 vector expressing hFIX | Right hepatic artery | Therapeutic levels of FIX attained for 2–4 weeks at the highest dose of administered vector. This dose was also associated with an immune response against transduced hepatocytes, abrogating clinical benefits |
[34] |
FIX: Factor IX; FVIII: Factor VIII; hFIX: Human factor IX; hFVIII: Human factor VIII; rAAV: Recombinant adeno-associated viral.