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- W4321181911 abstract "Sickle Cell Disease (SCD) is a monogenic disease caused by a nucleotide mutation in the β-globin gene. Current gene therapy studies are mainly focused on lentivirus vector-mediated gene addition or CRISPR/Cas9-mediated fetal globin reactivation, leaving the root cause unfixed. We developed a vectorized prime editing system that can directly repair the SCD mutation in hematopoietic stem cells (HSCs) in vivo in a SCD mouse model (CD46/Townes mice). Our approach involved a single intravenous injection of a non-integrating, prime editor-expressing virus vector into mobilized CD46/Townes mice and low-dose drug selection in vivo. This procedure resulted in the correction of ~40% of bS alleles in HSCs. On average 43% of HbS was replaced by HbA thereby greatly mitigating the SCD phenotypes. Transplantation in secondary recipients demonstrated that long-term repopulating HSCs were edited. Highly efficient target site editing was achieved with minimal generation of insertions and deletions and no detectable off-target editing. Because of its simplicity and portability, our in vivo prime editing approach has the potential for application in resource-poor countries where SCD is prevalent." @default.
- W4321181911 created "2023-02-18" @default.
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- W4321181911 date "2023-02-17" @default.
- W4321181911 modified "2023-10-11" @default.
- W4321181911 title "<i>In vivo</i> HSC prime editing rescues Sickle Cell Disease in a mouse model" @default.
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- W4321181911 doi "https://doi.org/10.1182/blood.2022018252" @default.
- W4321181911 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/36800642" @default.
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