Generalized-Gangliosidoses-Information-Page

Generalized Gangliosidoses Information Page


What research is being done?

Scientists are studying the mechanisms involving lipid buildup and resulting harm to the body. Research funded by the National Institute of Neurological Disorders and Stroke (NINDS) focuses on a better understandng of the diseases and on the development of new treatments targeting disease mechanisms. NINDS-funded research on the gangliosidoses is expanding the use of adeno-associated virus (AAV) delivered gene therapy to a larger area of the brain using an animal model of Tay-Sachs and Sandhoff diseases. A related project will study the effectivenss of whole-body AAV therapy in treating the disease.

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What research is being done?

Scientists are studying the mechanisms involving lipid buildup and resulting harm to the body. Research funded by the National Institute of Neurological Disorders and Stroke (NINDS) focuses on a better understandng of the diseases and on the development of new treatments targeting disease mechanisms. NINDS-funded research on the gangliosidoses is expanding the use of adeno-associated virus (AAV) delivered gene therapy to a larger area of the brain using an animal model of Tay-Sachs and Sandhoff diseases. A related project will study the effectivenss of whole-body AAV therapy in treating the disease.

Scientists are studying the mechanisms involving lipid buildup and resulting harm to the body. Research funded by the National Institute of Neurological Disorders and Stroke (NINDS) focuses on a better understandng of the diseases and on the development of new treatments targeting disease mechanisms. NINDS-funded research on the gangliosidoses is expanding the use of adeno-associated virus (AAV) delivered gene therapy to a larger area of the brain using an animal model of Tay-Sachs and Sandhoff diseases. A related project will study the effectivenss of whole-body AAV therapy in treating the disease.


Definition
Definition
Treatment
Treatment
Prognosis
Prognosis
Clinical Trials
Clinical Trials
Organizations
Organizations
Publications
Publications
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