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

(The Conversation is an independent and nonprofit source of news, analysis and commentary.

United statesUnited kingdomWarren tayBernard sachsAuburn universityOur translational institute for molecular therapeuticsUmass chan medical schoolChan medical schoolTranslational instituteMolecular therapeuticsCreative commonsBernard sachsMiguel sena estevesTtps theconversation com first gene therapy for tay sachs disease successfully given to two children 176870Tay sachsUmass chan medical school

2 Kids Get First Gene Therapy for Tay-Sachs Disease

And it was a success.

United statesUnited kingdomWarren tayBernard sachsAuburn universityOur translational institute for molecular therapeuticsUmass chan medical schoolChan medical schoolTranslational instituteMolecular therapeutics

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

Tay-Sachs is a rare and fatal neurodegerative disorder that most commonly affects children. Researchers have developed the first Tay-Sachs treatment to reach clinical trials.

United statesUnited kingdomWarren tayBernard sachsAuburn universityOur translational institute for molecular therapeuticsUmass chan medical schoolChan medical schoolTranslational instituteMolecular therapeuticsAdeno associated virusGene therapyHealth research fundingNeurodegenerative disordersRare diseasesResearch funding

2 Kids Get First Gene Therapy for Tay-Sachs Disease

And it was a success.

United statesUnited kingdomWarren tayBernard sachsAuburn universityOur translational institute for molecular therapeuticsUmass chan medical schoolChan medical schoolTranslational instituteMolecular therapeutics

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