Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy.
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L. Shihabuddin | Seng H. Cheng | M. Fiandaca | N. Boulis | M. Passini | J. Bu | Amy M. Richards | K. Bankiewicz | A. Kells | C. O’Riordan | T. Federici | Christopher M. Treleaven | A. Scaria | W. San Sebastián | J. Sullivan | L. Samaranch