Open Navigation
Close Navigation
Gene and cell therapy research shows promise for treating certain rare inherited diseases and forms of cancer.
Different forms of the disease being researched for potential treatments include CLN1, 2, 3, 5, 6, 7, and 8.
Gene therapy approaches target a faulty GLA gene.
Visit these pages for information on specific diseases and how gene therapy can help.
Take a closer look at beta thalassemia and Fanconi anemia.
Learn about approved treatments for blood cancers, such as leukemia and lymphoma.
Gene therapy has the potential to increase clotting factors to treat hemophilia A and B.
A genetic disorder caused by a breakdown of nerve cells in the brain.
A closer look at gene therapy for Leber congenital amaurosis (LCA), retinitis pigmentosa, choroideremia, Stargardt’s disease, and achromatopsia.
These are a set of rare genetic disorders including cerebral adrenoleukodystrophy (CALD), and metachromatic leukodystrophy (MLD).
Learn more about gene therapy research for mucopolysaccharidoses (MPS) types I, II, III and VI, also referred to as Hurler syndrome, Hunter syndrome, and Sanfilippo syndrome.
Gene therapy has the potential to treat Duchenne and certain subtypes of limb-girdle muscular dystrophy.
Learn about the potential to treat a degenerative neurological disease that over time causes the death of brain cells.
This rare genetic glycogen storage disease is caused by a faulty GAA gene.
Learn how gene therapy and gene editing may address the genetic cause of sickle cell disease.
Learn more about the FDA-approved gene therapy Zolgensma that targets the SMN1 gene.
Also known as XLMTM, this rare genetic disorder primarily causes muscle weakness caused by a faulty MTM1 gene.