Scientific, reliable information for patients and the public

Gene Therapy 101 Disease Treatments

Gene Therapy 101

Gene Therapy Basics

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.

Different Approaches

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?

Developing A Treatment

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.

Disease Treatments

Blood Cancers and CAR T-Cell Therapy

CAR T-cell therapy helps the immune system recognize cancer cells, like those in the blood cancers leukemia and lymphoma, as a threat and eliminate them.

Pompe Disease

Pompe disease is a genetic disorder that occurs when our bodies lack an important digestive enzyme called acid alpha-glucosidase, or GAA for short.


Hemophilia is a genetic disease that prevents blood from clotting properly leading to prolonged internal and external bleeding. 


Mucopolysaccharidoses (MPS) are a group of genetic disorders that affect our body’s ability to break down and recycle sugar molecules called glycosaminoglycans, or GAGs, leading to buildup and causing severe symptoms.

Muscular Dystrophy

Muscular dystrophy is a group of genetic diseases that cause progressive muscle weakness and loss of muscle mass. There are a wide variety of muscular dystrophies, each caused by different gene mutations.

Huntington's Disease

Huntington’s disease is a genetic disorder caused by a breakdown of nerve cells in the brain. The disease affects an individual’s ability to move, their mood, and how they think.

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.

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