DNA repair outcomes at nicks and mismatches introduced by base editors at the PCSK9 and HBG loci in human embryos are evaluated, finding that unlike Cas9-induced DNA breaks, base editor-induced lesions are efficiently repaired.
A detailed protocol for performing gene editing using an adenine base editor (ABE) to induce a single A-G transition mutation in a gene of therapeutic relevance is provided, enabling the production of cell models suited to the study of both dominant and recessive mutations.
Shun Zhang, B. P. Hubbard· Methods in molecular biology· 0 citations
An efficient Cas9d system (Cas9dUltra) is developed through gRNA and protein engineering, and its base editors (9dBEs) further developed through gRNA and protein engineering, enabling efficient and precise genome editing in human cells.
Qingquan Xiao, Zhijin Tian, Luqi Weng et al.· Advancement of science· 0 citations
Genome engineering is evolving from a DNA-cutting system to a precise and programmable genome editing system, which may support the future development of safer and more precise therapeutic strategies.
Vikash Kumar, Aman Kumar, Balak Das Kurmi· Current Pharmacogenomics and...· 0 citations
Genome editing in avian systems has largely relied on plasmid-based CRISPR systems and antibiotic selection to achieve successful editing. However, plasmid delivery can result in prolonged nuclease expression and potential unintended DNA integration. In addition, selection-based enrichment may mask the intrinsic geno...
Sydney G. Bingham, Jin Lee Kim, Kiho Lee et al.· Journal of Animal Science· 0 citations
De novo mutations are a major source of genetic variation and disease risk, yet the developmental timing and mechanisms underlying their origin require further investigation. While germ cells have traditionally been considered the primary source of these mutations, increasing evidence suggests that a substantial fracti...
M. P. Grejo, J. Augusto, Angélica C. Dos Santos et al.· Molecular human reproduction· 0 citations
Findings indicate that the Cas12a system does not yield locus excision at the efficiency required for sensitive negative selection assays, and showed no meaningful correlation between gRNA efficiency, orientation, GC content, or cut site geometry and the success of locus excision.
N. Anvar, Zsofia M. Szegletes, R. Steger et al.· bioRxiv· 0 citations
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