Prime editing for precise and highly versatile genome manipulation
Programmable gene-editing tools have transformed the life sciences and have shown
potential for the treatment of genetic disease. Among the CRISPR–Cas technologies that …
potential for the treatment of genetic disease. Among the CRISPR–Cas technologies that …
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 …
Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing
CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …
decade and holds great promise for the treatment of human genetic diseases. Here we …
[HTML][HTML] Massively parallel base editing to map variant effects in human hematopoiesis
JD Martin-Rufino, N Castano, M Pang, EI Grody… - Cell, 2023 - cell.com
Systematic evaluation of the impact of genetic variants is critical for the study and treatment
of human physiology and disease. While specific mutations can be introduced by genome …
of human physiology and disease. While specific mutations can be introduced by genome …
Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells
Prime editing (PE) enables precise and versatile genome editing without requiring double-
stranded DNA breaks. Here we describe the systematic optimization of PE systems to …
stranded DNA breaks. Here we describe the systematic optimization of PE systems to …
[HTML][HTML] Emergent CRISPR–Cas-based technologies for engineering non-model bacteria
Highlights•CRISPR–Cas systems promoted the engineering of non-model bacteria.•Tools
include genome modifications and finely-tuned transcriptional regulation.•CRISPR–Cas …
include genome modifications and finely-tuned transcriptional regulation.•CRISPR–Cas …
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy
The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …
LINE-1 retrotransposition and its deregulation in cancers: implications for therapeutic opportunities
C Mendez-Dorantes, KH Burns - Genes & development, 2023 - genesdev.cshlp.org
Abstract Long interspersed element 1 (LINE-1) is the only protein-coding transposon that is
active in humans. LINE-1 propagates in the genome using RNA intermediates via …
active in humans. LINE-1 propagates in the genome using RNA intermediates via …
Non-viral approaches in CAR-NK cell engineering: connecting natural killer cell biology and gene delivery
EM McErlean, HO McCarthy - Journal of Nanobiotechnology, 2024 - Springer
Natural Killer (NK) cells are exciting candidates for cancer immunotherapy with potent innate
cytotoxicity and distinct advantages over T cells for Chimeric Antigen Receptor (CAR) …
cytotoxicity and distinct advantages over T cells for Chimeric Antigen Receptor (CAR) …
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering
S Eghbalsaied, C Lawler, B Petersen, RA Hajiyev… - Gene Therapy, 2024 - nature.com
Base editors are a type of double-stranded break (DSB)-free gene editing technology that
has opened up new possibilities for precise manipulation of mitochondrial DNA (mtDNA) …
has opened up new possibilities for precise manipulation of mitochondrial DNA (mtDNA) …