Tag: <span>Tay-Sachs disease</span>

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First gene therapy for Tay-Sachs disease successfully given to two children
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First gene therapy for Tay-Sachs disease successfully given to two children

by Miguel Sena-Esteves, The Conversation Credit: Pixabay/CC0 Public Domain Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development. Tay-Sachs is a severe neurological disease caused by a deficiency in an enzyme called HexA. This enzyme breaks down a fatlike substance that normally exists in very small, harmless amounts in the brain. Without...

Cord blood cell transplantation and curcumin administration tested as therapy of Tay-Sachs disease
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Cord blood cell transplantation and curcumin administration tested as therapy of Tay-Sachs disease

KAZAN FEDERAL UNIVERSITY IMAGE: (A) DYNAMIC CHANGES IN THE HEXA ACTIVITY IN THE TSD PATIENT’S PLASMA AFTER UCBCT AND CURCUMIN ADMINISTRATION; NORMAL LEVEL OF HEXA ACTIVITY OBSERVED IN HEALTHY DONORS IS 24–148 NMOL/ML/H; (B) DYNAMIC CHANGES IN THE HEXA CONCENTRATION IN THE TSD PATIENT’S SERUM AFTER UCBCT AND CURCUMIN ADMINISTRATION. THE HEXA CONCENTRATION RANGE MEASURED...