Gene and Cell Therapy Clinical Trials

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Acute Lymphoblastic Leukemia

Acute myeloid leukemia

Beta thalassemia

Choroideremia

Hemophilia A

Hemophilia B

Leber congenital amaurosis

Methylmalonic acidemia (MMA)

MPS I

MPS II (Hunter syndrome)

Non-hodgkin lymphoma

Phenylketonurias

Sickle cell disease

Spinal muscular atrophy