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New hope for older SMA patients with next-generation self-complementary AAV gene therapy

Hiroyuki Nakai1, Erika L Finanger2, Richard Jude Samulski3

  • 1Department of Molecular and Medical Genetics, Oregon Health & Science University School of Medicine, Portland, OR, USA; Department of Molecular Microbiology and Immunology, Oregon Health & Science University School of Medicine, Portland, OR, USA; Division of Neuroscience, Oregon National Primate Research Center, Beaverton, OR, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|September 23, 2025
PubMed
Summary

No abstract available in PubMed .

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