In vivo somatic cell base editing and prime editing
Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …
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 …
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice
Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition
Therapeutic applications of nuclease-based genome editing would benefit from improved
methods for transgene integration via homology-directed repair (HDR). To improve HDR …
methods for transgene integration via homology-directed repair (HDR). To improve HDR …
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
In vivo HSC prime editing rescues sickle cell disease in a mouse model
Sickle cell disease (SCD) is a monogenic disease caused by a nucleotide mutation in the β-
globin gene. Current gene therapy studies are mainly focused on lentiviral vector–mediated …
globin gene. Current gene therapy studies are mainly focused on lentiviral vector–mediated …
Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing
Most genome editing analyses to date are based on quantifying small insertions and
deletions. Here, we show that CRISPR-Cas9 genome editing can induce large gene …
deletions. Here, we show that CRISPR-Cas9 genome editing can induce large gene …
Successes and challenges in clinical gene therapy
Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …
continued to advance and provide enduring treatments for increasing number of diseases …
Sickle cell disease: from genetics to curative approaches
G Hardouin, E Magrin, A Corsia… - Annual Review of …, 2023 - annualreviews.org
Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …
Recent advances in CRISPR-based genome editing technology and its applications in cardiovascular research
ZH Li, J Wang, JP Xu, J Wang, X Yang - Military Medical Research, 2023 - Springer
The rapid development of genome editing technology has brought major breakthroughs in
the fields of life science and medicine. In recent years, the clustered regularly interspaced …
the fields of life science and medicine. In recent years, the clustered regularly interspaced …