A severe inherited neuromuscular disease is corrected in mice by intravenous gene delivery.
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Change history
06 October 2022
Editor’s Note: readers are alerted that the primary research paper covered in this News & Views has been retracted.
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MacKenzie, A. Genetic therapy for spinal muscular atrophy. Nat Biotechnol 28, 235–237 (2010). https://doi.org/10.1038/nbt0310-235
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DOI: https://doi.org/10.1038/nbt0310-235
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