This phase 1/2 study will determine the safety, feasibility, and efficacy of CD33 CART cells following lymphodepleting chemotherapy in patients with acute myeloid leukemia (AML).

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa)

This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver.

Individuals with a certain type of beta thalassemia that is treated with regular transfusions, and who are 50 years old and younger, may be able to participate.

This purpose of this study is to determine the efficacy and safety of tisagenlecleucel (also referred to as CTL019, or Kymriah™) in children, adolescents and young adults with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL).

This purpose of this study is to determine the efficacy of humanized CD19 CAR  T cells (huCART19) in pediatric and young adult patients with high-risk relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).

This study will take the patients white blood cells - T cells - and change them to turn against the cancer.

This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19.

This study will evaluate the safety and tolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with a neurocognitive deficit that are at least 4 months old.

The purpose of this study is to look at the safety and tolerability of RBX-121, a one-time gene therapy, in subjects with MPS II (Hunter Syndrome).