William Peranteau

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Prenatal editing halts progression of Hurler syndrome in pre-clinical model

US researchers used an AAV9 vector to edit a single…

14 July 2021 | By Anna Begley (Drug Target Review)

US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.

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William Peranteau

Prenatal editing halts progression of Hurler syndrome in pre-clinical model