Allele-specific gene editing to rescue dominant crx-associated lca7 phenotypes in a retinal organoid model

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• who: Kathleen R. Chirco from the University of California San Francisco, San Francisco, CA, USA have published the article: Allele-specific gene editing to rescue dominant CRX-associated LCA7 phenotypes in a retinal organoid model, in the Journal: (JOURNAL)
• what: This work provides early evidence for an effective approach to treat LCA7 which can be applied more broadly to other dominant genetic diseases. The authors aimed to establish an in_vitro human retinal organoid model system to study mutation-specific LCA7 phenotypes, allowing the authors to visualize the initiation and progression of disease phenotypes as the . . .
  

   

  

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