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Updated: Feb 20, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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血管新生幹細胞遺伝子療法によるシスティノシス治療

Bruce A Barshop1, Edward D Ball2, Nadine Benador3,4

  • 1Department of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.

The New England journal of medicine
|February 18, 2026
PubMed
まとめ

この研究では,システィノシスに対するCTNS-RD-04遺伝子治療を評価し,治療後の白血球システィン濃度の低下を示した. 副作用は主に軽度で,処置や疾患に関連していた.

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