An AAV9 Encoding Human ABCD1 (SBT101) Shows Functional Improvement Following Spinal Cord Delivery in a Rodent Model of Adrenomyeloneuropathy

A. Pujol,A. Bosch,I. M. E. Dijkstra,S. Kemp,Y. R. J. Jaspers, S. Verdes, S. Fourcade, C. Guilera, M. Leal-Julia, A. Onieva, V. Vasireddy, S. W. Clark, D. W. Anderson,K. Kozarsky

MOLECULAR THERAPY(2022)

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