Microhomology-Mediated End Joining Chronicles: Tracing the Evolutionary Footprints of Genome Protection
A Sfeir, M Tijsterman, M McVey - Annual Review of Cell and …, 2024 - annualreviews.org
The fidelity of genetic information is essential for cellular function and viability. DNA double-
strand breaks (DSBs) pose a significant threat to genome integrity, necessitating efficient …
strand breaks (DSBs) pose a significant threat to genome integrity, necessitating efficient …
Recent advances in CRISPR/Cas-based genome insertion technologies
X Chen, J Du, S Yun, C Xue, Y Yao, S Rao - Molecular Therapy-Nucleic …, 2024 - cell.com
Programmable genome insertion (or knock-in) is vital for both fundamental and translational
research. The continuously expanding number of CRISPR-based genome insertion …
research. The continuously expanding number of CRISPR-based genome insertion …
A Type II-B Cas9 nuclease with minimized off-targets and reduced chromosomal translocations in vivo
B Bestas, S Wimberger, D Degtev, A Madsen… - Nature …, 2023 - nature.com
Abstract Streptococcus pyogenes Cas9 (SpCas9) and derived enzymes are widely used as
genome editors, but their promiscuous nuclease activity often induces undesired mutations …
genome editors, but their promiscuous nuclease activity often induces undesired mutations …
On-and off-target effects of paired CRISPR-Cas nickase in primary human cells
J Klermund, M Rhiel, T Kocher, KO Chmielewski… - Molecular Therapy, 2024 - cell.com
Undesired on-and off-target effects of CRISPR-Cas nucleases remain a challenge in
genome editing. While the use of Cas9 nickases has been shown to minimize off-target …
genome editing. While the use of Cas9 nickases has been shown to minimize off-target …
Deficiency of both classical and alternative end‐joining pathways leads to a synergistic defect in double‐strand break repair but not to an increase in homology …
L Merker, L Feller, A Dorn, H Puchta - The Plant Journal, 2024 - Wiley Online Library
In eukaryotes, double‐strand breaks (DSBs) are either repaired by homologous
recombination (HR) or non‐homologous end‐joining (NHEJ). In somatic plant cells, HR is …
recombination (HR) or non‐homologous end‐joining (NHEJ). In somatic plant cells, HR is …
Magnetic Nanoparticle‐Assisted Non‐Viral CRISPR‐Cas9 for Enhanced Genome Editing to Treat Rett Syndrome
HY Cho, M Yoo, T Pongkulapa, H Rabie… - Advanced …, 2024 - Wiley Online Library
The CRISPR‐Cas9 technology has the potential to revolutionize the treatment of various
diseases, including Rett syndrome, by enabling the correction of genes or mutations in …
diseases, including Rett syndrome, by enabling the correction of genes or mutations in …
Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells
A Moiani, G Letort, S Lizot, A Chalumeau… - Nature …, 2024 - nature.com
Sickle cell disease is a devastating blood disorder that originates from a single point
mutation in the HBB gene coding for hemoglobin. Here, we develop a GMP-compatible …
mutation in the HBB gene coding for hemoglobin. Here, we develop a GMP-compatible …
Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells
SE Luna, J Camarena, JP Hampton… - Nature Biomedical …, 2024 - nature.com
Some gene polymorphisms can lead to monogenic diseases, whereas other polymorphisms
may confer beneficial traits. A well-characterized example is congenital erythrocytosis—the …
may confer beneficial traits. A well-characterized example is congenital erythrocytosis—the …
Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders
D Allen, N Kalter, M Rosenberg, A Hendel - Pharmaceutics, 2023 - mdpi.com
Genome engineering via targeted nucleases, specifically CRISPR-Cas9, has revolutionized
the field of gene therapy research, providing a potential treatment for diseases of the blood …
the field of gene therapy research, providing a potential treatment for diseases of the blood …
Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair
JA Perez-Bermejo, O Efagene, WM Matern… - Nature …, 2024 - nature.com
Abstract Homology Directed Repair (HDR) enables precise genome editing, but the
implementation of HDR-based therapies is hindered by limited efficiency in comparison to …
implementation of HDR-based therapies is hindered by limited efficiency in comparison to …