[HTML][HTML] Genotoxic effects of base and prime editing in human hematopoietic stem cells

M Fiumara, S Ferrari, A Omer-Javed, S Beretta… - Nature …, 2024 - nature.com
Base and prime editors (BEs and PEs) may provide more precise genetic engineering than
nuclease-based approaches because they bypass the dependence on DNA double-strand …

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors

JL Doman, A Raguram, GA Newby, DR Liu - Nature biotechnology, 2020 - nature.com
Cytosine base editors (CBEs) enable targeted C• G-to-T• A conversions in genomic DNA.
Recent studies report that BE3, the original CBE, induces a low frequency of genome-wide …

[HTML][HTML] Guide RNA engineering enables efficient CRISPR editing with a miniature Syntrophomonas palmitatica Cas12f1 nuclease

Y Wang, Y Wang, D Pan, H Yu, Y Zhang, W Chen, F Li… - Cell Reports, 2022 - cell.com
Gene therapy is limited by inefficient delivery of large clustered regularly interspaced short
palindromic repeat (CRISPR) effectors, such as Cas9 and Cas12a nucleases. Cas12f …

Impact of CRISPR/HDR editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques

BC Lee, A Gin, C Wu, K Singh, M Grice, R Mortlock… - Cell stem cell, 2024 - cell.com
For precise genome editing via CRISPR/homology-directed repair (HDR), effective and safe
editing of long-term engrafting hematopoietic stem cells (LT-HSCs) is required. The impact …

[HTML][HTML] Coiled-coil heterodimer-based recruitment of an exonuclease to CRISPR/Cas for enhanced gene editing

D Lainšček, V Forstnerič, V Mikolič, Š Malenšek… - Nature …, 2022 - nature.com
The CRISPR/Cas system has emerged as a powerful and versatile genome engineering
tool, revolutionizing biological and biomedical sciences, where an improvement of efficiency …

[HTML][HTML] Small molecule inhibition of ATM kinase increases CRISPR-Cas9 1-bp insertion frequency

HC Bermudez-Cabrera, S Culbertson, S Barkal… - Nature …, 2021 - nature.com
Mutational outcomes following CRISPR-Cas9-nuclease cutting in mammalian cells have
recently been shown to be predictable and, in certain cases, skewed toward single …

Heritable, multinucleotide deletions in plants using viral delivery of a repair exonuclease and guide RNAs

D Liu, EA Myers, S Xuan, LE Prichard… - Plant …, 2024 - academic.oup.com
CRISPR/Cas9-mediated mutagenesis typically results in short insertion/deletion mutations,
which are often too small to disrupt the function of cis-acting regulatory elements. Here, we …

[HTML][HTML] AAV-CRISPR/Cas9 gene editing preserves long-term vision in the P23H rat model of autosomal dominant retinitis pigmentosa

S Shahin, H Xu, B Lu, A Mercado, MK Jones… - Pharmaceutics, 2022 - mdpi.com
Retinitis pigmentosa (RP) consists of a group of inherited, retinal degenerative disorders
and is characterized by progressive loss of rod photoreceptors and eventual degeneration of …

Combined theoretical, bioinformatic, and biochemical analyses of RNA editing by adenine base editors

KL Rallapalli, BL Ranzau, KR Ganapathy… - The CRISPR …, 2022 - liebertpub.com
Adenine base editors (ABEs) have been subjected to multiple rounds of mutagenesis with
the goal of optimizing their function as efficient and precise genome editing agents. Despite …

[HTML][HTML] Improved loss-of-function CRISPR-Cas9 genome editing in human cells concomitant with inhibition of TGF-β signaling

T Mishra, V Bhardwaj, N Ahuja, P Gadgil… - … Therapy-Nucleic Acids, 2022 - cell.com
Strategies to modulate cellular DNA repair pathways hold immense potential to enhance the
efficiency of CRISPR-Cas9 genome editing platform. In the absence of a repair template …