[HTML][HTML] The CRISPR-Cas toolbox and gene editing technologies

G Liu, Q Lin, S Jin, C Gao - Molecular cell, 2022 - cell.com
The emergence of CRISPR-Cas systems has accelerated the development of gene editing
technologies, which are widely used in the life sciences. To improve the performance of …

CRISPR-based therapeutics: current challenges and future applications

AE Modell, D Lim, TM Nguyen, V Sreekanth… - Trends in …, 2022 - cell.com
The discovery, only a decade ago, of the genome editing power of clustered regularly
interspaced short palindromic repeats (CRISPR)-associated nucleases is already …

Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors

JR Davis, X Wang, IP Witte, TP Huang… - Nature Biomedical …, 2022 - nature.com
The viral delivery of base editors has been complicated by their size and by the limited
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …

A highly specific CRISPR-Cas12j nuclease enables allele-specific genome editing

Y Wang, T Qi, J Liu, Y Yang, Z Wang, Y Wang… - Science …, 2023 - science.org
The CRISPR-Cas system can treat autosomal dominant diseases by nonhomologous end
joining (NHEJ) gene disruption of mutant alleles. However, many single-nucleotide …

Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

R Ibraheim, PWL Tai, A Mir, N Javeed, J Wang… - Nature …, 2021 - nature.com
Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic
genome editing but are severely constrained by cargo limits. Simultaneous delivery of …

Continuous directed evolution of a compact CjCas9 variant with broad PAM compatibility

L Schmidheini, N Mathis, KF Marquart… - Nature Chemical …, 2024 - nature.com
CRISPR–Cas9 genome engineering is a powerful technology for correcting genetic
diseases. However, the targeting range of Cas9 proteins is limited by their requirement for a …

A split and inducible adenine base editor for precise in vivo base editing

H Zeng, Q Yuan, F Peng, D Ma, A Lingineni… - Nature …, 2023 - nature.com
DNA base editors use deaminases fused to a programmable DNA-binding protein for
targeted nucleotide conversion. However, the most widely used TadA deaminases lack post …

Global detection of DNA repair outcomes induced by CRISPR–Cas9

M Liu, W Zhang, C Xin, J Yin, Y Shang, C Ai… - Nucleic acids …, 2021 - academic.oup.com
CRISPR–Cas9 generates double-stranded DNA breaks (DSBs) to activate cellular DNA
repair pathways for genome editing. The repair of DSBs leads to small insertions or …

Hypercompact adenine base editors based on a Cas12f variant guided by engineered RNA

DY Kim, Y Chung, Y Lee, D Jeong, KH Park… - Nature chemical …, 2022 - nature.com
Cas12f is a hypercompact type V, Cas12 family member. Previously, we reported a set of
engineered guide RNAs supporting high indel efficiency for Cas12f1 in human cells. Here …

Rationally designed base editors for precise editing of the sickle cell disease mutation

SH Chu, M Packer, H Rees, D Lam, Y Yu… - The CRISPR …, 2021 - liebertpub.com
Base editors are fusions of a deaminase and CRISPR-Cas ribonucleoprotein that allow
programmable installment of transition mutations without double-strand DNA break …