4.1 Editorial Material

How far away is spinal muscular atrophy gene therapy?

Journal

EXPERT REVIEW OF NEUROTHERAPEUTICS
Volume 15, Issue 9, Pages 965-968

Publisher

TAYLOR & FRANCIS LTD
DOI: 10.1586/14737175.2015.1070672

Keywords

AAV9; delivery route; gene therapy; SMN; spinal muscular atrophy; therapeutic time window

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