Gene Therapy With More Effective Carrier Shines in Mouse Study

Gene Therapy With More Effective Carrier Shines in Mouse Study

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Gene Therapy With More Effective Carrier Shines in Mouse Study

A gene therapy delivered through a more effective viral carrier safely and effectively corrected enzymatic deficiency, restored cognitive and motor function, and improved survival in a mouse model of Sanfilippo syndrome type A, a study shows. The findings suggest this may be a more effective gene therapy approach to prevent and reverse the broad symptoms of this condition, and support its evaluation in future clinical trials, the researchers noted. The study, “Targeting the Root Cause…

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