Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
CRISPR/Cas9 therapeutics: progress and prospects
T Li, Y Yang, H Qi, W Cui, L Zhang, X Fu, X He… - … and Targeted Therapy, 2023 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …
Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity
Cytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …
Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors
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 …
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …
Drug delivery systems for CRISPR-based genome editors
V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …
these drugs must enter the desired cell without eliciting an unwanted immune response, so …
CRISPR technologies and the search for the PAM-free nuclease
D Collias, CL Beisel - Nature communications, 2021 - nature.com
The ever-expanding set of CRISPR technologies and their programmable RNA-guided
nucleases exhibit remarkable flexibility in DNA targeting. However, this flexibility comes with …
nucleases exhibit remarkable flexibility in DNA targeting. However, this flexibility comes with …
Continuous evolution of SpCas9 variants compatible with non-G PAMs
The targeting scope of Streptococcus pyogenes Cas9 (SpCas9) and its engineered variants
is largely restricted to protospacer-adjacent motif (PAM) sequences containing G bases …
is largely restricted to protospacer-adjacent motif (PAM) sequences containing G bases …
Engineered CRISPR-OsCas12f1 and RhCas12f1 with robust activities and expanded target range for genome editing
X Kong, H Zhang, G Li, Z Wang, X Kong, L Wang… - Nature …, 2023 - nature.com
Abstract The type VF CRISPR-Cas12f system is a strong candidate for therapeutic
applications due to the compact size of the Cas12f proteins. In this work, we identify six …
applications due to the compact size of the Cas12f proteins. In this work, we identify six …
High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs
TP Huang, ZJ Heins, SM Miller, BG Wong… - Nature …, 2023 - nature.com
Despite the availability of Cas9 variants with varied protospacer-adjacent motif (PAM)
compatibilities, some genomic loci—especially those with pyrimidine-rich PAM sequences …
compatibilities, some genomic loci—especially those with pyrimidine-rich PAM sequences …