Cell and Gene Therapy Clinical Trials

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