No abstract available
Keywords:
AAV9; ISS-N1; Spinraza; gene therapy; nusinersen; oligonucleotide; spinal muscular atrophy (SMA); survival motor neuron (SMN).
Publication types
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Editorial
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
MeSH terms
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Exodeoxyribonucleases / genetics
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Genetic Therapy
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Humans
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Muscular Atrophy, Spinal / genetics
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Muscular Atrophy, Spinal / therapy*
Substances
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DCLRE1B protein, human
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Exodeoxyribonucleases