Gene Therapy 101 Disease Treatments
Gene therapy has been studied for more than 40 years and can help stop or slow the effects of disease on the most basic level of the human body—our genes. And to understand how it works, we’ll start at the basics.
Gene therapy, cell therapy and gene editing are fields with a similar goal: To treat disease by changing our bodies at a microscopic level. What do these approaches have in common, and what makes them different?
Gene therapies take years to go from theoretical concepts, to preclinical trials, to clinical trials, and, finally, into actual treatments that can improve the lives of patients. Here we learn about the process of developing a treatment.
Gene therapy to treat SMA is now approved for clinical use in the United States.
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.
Gene therapies for sickle cell disease, beta-thalessemia, and Fanconi anemia all show promise and are at various stages of clinical trials.
Among the most developed gene therapies, Learn more about available treatments and others on the horizon for LCA, retinitis pegmentosa, choroideremia and more.
Additional websites, information, definitions and FAQs for patients, families, advocates, and the public.
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