Article
CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.
Science translational medicine - 11 Jan 2017
De Ravin Suk See, Li Linhong, Wu Xiaolin, Choi Uimook, Allen Cornell, Koontz Sherry, Lee Janet, Theobald-Whiting Narda, Chu Jessica, Garofalo Mary, Sweeney Colin, Kardava Lela, Moir Susan, Viley Angelia, Natarajan Pachai, Su Ling, Kuhns Douglas, Zarember Kol A, Peshwa Madhusudan V, Malech Harry L
Abstract excerpt
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...
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