Disease Treatments

ASGCT on SciShow

ASGCT partnered with SciShow to discuss three groundbreaking gene therapy frontiers. These videos are a great complement to ASGCT's Patient Education portal and we're proud to support free educational community resources. Watch all three episodes on YouTube!

Parkinson's Disease

Parkinson’s degenerative neurological diseaseis a genetic disease that affects 1 in 100 people over the age of 60 and, over time, causes the death of brain cells, called neurons. However, gene therapy might offer hope for managing symptoms, or even improving the effects of existing medications.

Spinal Muscular Atrophy

Gene therapy to treat SMA is now approved for clinical use in the United States.

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 Diseases

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.


ASGCT Policy Summit

September 26-27, 2022

This site uses cookies to offer you a better user experience and to analyze site traffic. By continuing to use this website, you accept our use of cookies.