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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.
Learn how in vivo and ex vivo gene therapy approaches can be used to treat Fabry disease by delivering a working GLA gene into cells.
Learn about this rare genetic disorder that causes the harmful buildup of glycolipids throughout the body and how gene therapy may be able to address its symptoms.
Gene therapy for GM1 aims to be a one-time treatment that delivers a working copy of the GLB1 gene into cells using a viral vector.
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.
Gene therapy may target the cause of this disease, which causes excess copper to be stored in the body.
Also known as XLMTM, this rare genetic disorder primarily causes muscle weakness caused by a faulty MTM1 gene.