| Disease |
Affected tissue |
Target cell |
Type of vector |
Reference |
Chronic
Granulomatous
Disease (CGD) |
Hematopoietic System, Immunodeficiency |
CD34+ hematopoietic stem/progenitor cells |
Retroviral (oncoretroviral) |
30 |
| Adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) |
Hematopoietic System, Immunodeficiency |
CD34+ hematopoietic stem/progenitor cells |
Retroviral (oncoretroviral) |
31 |
| X-linked severe combined immunodeficiency (X-SCID) |
Hematopoietic System, Immunodeficiency |
CD34+ hematopoietic stem/progenitor cells |
Retroviral (oncoretroviral) |
24 |
| β-thalassemia |
Hematopoietic System, Ineffective erythropoiesis |
CD34+ hematopoietic stem/progenitor cells |
Retroviral (lentiviral) |
28 |
| X-linked adrenoleukodystrophy (ALD) |
Brain |
CD34+ hematopoietic stem/progenitor cells |
Retroviral (lentiviral) |
23 |
| Metachromatic leukodystrophy (MLD) |
Brain |
CD34+ hematopoietic stem/progenitor cells |
Retroviral (lentiviral) |
33 |
