Enhancing homology-directed repair efficiency with HDR-boosting modular ssDNA donor
Despite the potential of small molecules and recombinant proteins to enhance the efficiency
of homology-directed repair (HDR), single-stranded DNA (ssDNA) donors, as currently …
of homology-directed repair (HDR), single-stranded DNA (ssDNA) donors, as currently …
Recent advances in CRISPR/Cas-based genome insertion technologies
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 …
Genome editing with the HDR-enhancing DNA-PKcs inhibitor AZD7648 causes large-scale genomic alterations
Abstract The DNA-PKcs inhibitor AZD7648 enhances CRISPR–Cas9-directed homology-
directed repair efficiencies, with potential for clinical utility, but its possible on-target …
directed repair efficiencies, with potential for clinical utility, but its possible on-target …
Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches
AF Leal, AM Herreno-Pachón… - International Journal of …, 2024 - mdpi.com
Since its discovery in 2012, the clustered regularly interspaced short palindromic repeats
(CRISPR) and CRISPR-associated protein 9 (Cas9) system has supposed a promising …
(CRISPR) and CRISPR-associated protein 9 (Cas9) system has supposed a promising …
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
MC Castiello, C Brandas, S Ferrari… - Science translational …, 2024 - science.org
Recombination activating genes (RAGs) are tightly regulated during lymphoid differentiation,
and their mutations cause a spectrum of severe immunological disorders. Hematopoietic …
and their mutations cause a spectrum of severe immunological disorders. Hematopoietic …
[HTML][HTML] DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome
Targeted gene editing to restore CD40L expression via homology-directed repair (HDR) in
CD34+ hematopoietic stem and progenitor cells (HSPCs) represents a potential long-term …
CD34+ hematopoietic stem and progenitor cells (HSPCs) represents a potential long-term …
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 …
Characterization of the role of spatial proximity of DNA double-strand breaks in the formation of CRISPR-Cas9-induced large structural variations
M Dahl-Jessen, T Terkelsen, RO Bak… - Genome …, 2025 - genome.cshlp.org
Structural variations (SVs) play important roles in genetic diversity, evolution, and
carcinogenesis and are, as such, important for human health. However, it remains unclear …
carcinogenesis and are, as such, important for human health. However, it remains unclear …
Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis
Blood transfusion plays a vital role in modern medicine, but frequent shortages occur. Ex
vivo manufacturing of red blood cells (RBCs) from universal donor cells offers a potential …
vivo manufacturing of red blood cells (RBCs) from universal donor cells offers a potential …
Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells
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 …