CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

@article{Ravin2017CRISPRCas9GR,
  title={CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease},
  author={Suk See De Ravin and Linhong Li and Xiaolin Wu and Uimook Choi and Cornell R. Allen and Sherry M. Koontz and Janet S. Lee and Narda L Theobald-Whiting and Jessica Chu and Mary Garofalo and Colin L. Sweeney and Lela Kardava and Susan Moir and Angelia M. Viley and Pachai Natarajan and Ling Yu Su and Douglas B. Kuhns and Kol A. Zarember and Madhusudan V. Peshwa and Harry L Malech},
  journal={Science Translational Medicine},
  year={2017},
  volume={9}
}
Gene repair of CD34+ hematopoietic stem and progenitor cells (HSPCs) may avoid problems associated with gene therapy, such as vector-related mutagenesis and dysregulated transgene expression. We used CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9 (CRISPR-associated 9) to repair a mutation in the CYBB gene of CD34+ HSPCs from patients with the immunodeficiency disorder X-linked chronic granulomatous disease (X-CGD). Sequence-confirmed repair of >20% of HSPCs from X-CGD… CONTINUE READING
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