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GENE THERAPY

A combined stem-cell–gene therapy strategy for ALS

Nature Medicine volume 28, pages 1751–1752 (2022)Cite this article

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A gene therapy product combines astrocyte replacement with stable delivery of a neuroprotective factor; a first-in-human study demonstrated safety and will inform the design of further clinical trials for this neurodegenerative disease.

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Authors and Affiliations

  1. Department of Physiology, Keio University School of Medicine, Shinjuku-ku, Tokyo, Japan

    Hideyuki Okano

  2. Department of Brain & Cognitive Sciences, McGovern Institute for Brain Research, Massachusetts Institute of Technologies, Cambridge, MA, USA

    Hideyuki Okano

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  1. Hideyuki Okano
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Correspondence to Hideyuki Okano.

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Competing interests

The author is a founding scientist of SanBio Co. Ltd. and K Pharma Inc.

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Okano, H. A combined stem-cell–gene therapy strategy for ALS. Nat Med 28, 1751–1752 (2022). https://doi.org/10.1038/s41591-022-01983-0

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