Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice
Hannah G. Lahey(University of Massachusetts Chan Medical School), Miguel Sena‐Esteves(University of Massachusetts Chan Medical School), Erin Horn(University of Massachusetts Chan Medical School), Diane Golebiowski(University of Massachusetts Chan Medical School), C Izzo(University of Massachusetts Chan Medical School), Misako Hwang(Auburn University), Lauren E. Ellis(Auburn University), Chelsea Joy Webber(Boston University), Heather Gray‐Edwards(UMass Memorial Health Care), Toloo Taghian(University of Massachusetts Chan Medical School), Paola Rodrı́guez(Universidad de la República de Uruguay), Ana Rita Batista(University of Massachusetts Chan Medical School), Douglas R. Martin(Auburn University)
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