Disease Treatments

ASGCT on SciShow

ASGCT is proud to partner with SciShow on a series of educational gene therapy videos. The first, Gene Therapy for Blood Disorders, is now available for free on YouTube!

Spinal Muscular Atrophy

On the verge of clinical approval, gene therapy for spinal muscle atrophy is pending FDA review.

X-Linked Myotubular Myopathy

The full story on the first gene therapy clinical trials for X-Linked MTM.


Gene therapy for ALD, MLD and other leukodystrophy diagnoses are in the early stages, but preclinical studies have been promising.

Blood Disorders

Gene therapies for sickle cell disease, beta-thalessemia, and Fanconi anemia all show promise and are at various stages of clinical trials.

Inherited Retinal Disorders

Among the most developed gene therapies, Learn more about available treatments and others on the horizon for LCA, retinitis pegmentosa, choroideremia and more.

More Resources

Additional websites, information, definitions and FAQs for patients, families, advocates, and the public.

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