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- W3119787031 endingPage "148" @default.
- W3119787031 startingPage "148" @default.
- W3119787031 abstract "There are an estimated 10 000 monogenic diseases affecting tens of millions of individuals worldwide. The application of CRISPR/Cas genome editing tools to treat monogenic diseases is an emerging strategy with the potential to generate personalized treatment approaches for these patients. CRISPR/Cas-based systems are programmable and sequence-specific genome editing tools with the capacity to generate base pair resolution manipulations to DNA or RNA. The complexity of genomic insults resulting in heritable disease requires patient-specific genome editing strategies with consideration of DNA repair pathways, and CRISPR/Cas systems of different types, species, and those with additional enzymatic capacity and/or delivery methods. In this review we aim to discuss broad and multifaceted therapeutic applications of CRISPR/Cas gene editing systems including in harnessing of homology directed repair, non-homologous end joining, microhomology-mediated end joining, and base editing to permanently correct diverse monogenic diseases." @default.
- W3119787031 created "2021-01-18" @default.
- W3119787031 creator A5055211375 @default.
- W3119787031 creator A5061170419 @default.
- W3119787031 date "2021-01-01" @default.
- W3119787031 modified "2023-10-16" @default.
- W3119787031 title "Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases" @default.
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