Results 61 to 70 of about 869,701 (314)

Gene Editing in Fabry Disease: A Strategy Delineation [PDF]

, 2022
Oral presentation by first authorThe use of iPSCs, in the last years became wide spread, even in our group at INSA, the use of iPSCs to develop models of disease is now envisaged for various Lysosomal Storage Diseases.
Moreira, Luciana, Amaral, Olga, Duarte, Ana J., Ribeiro, Diogo   +3 more
core  

Recruitment and rejoining of remote double-strand DNA breaks for enhanced and precise chromosome editing

Genome Biology
Chromosomal rearrangements, such as translocations, deletions, and inversions, underlie numerous genetic diseases and cancers, yet precise engineering of these rearrangements remains challenging.
Mingyao Wang, Pengchong Fu, Ziheng Chen, Xiangnan Wang, Hanhui Ma, Xuedi Zhang, Guanjun Gao   +6 more
doaj   +1 more source

Natural Killer Cells in Paediatric Soft Tissue Sarcomas: A Systematic Review

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Paediatric soft tissue sarcomas (pSTS) are a rare and heterogeneous group of malignant tumours arising in tissues of mesenchymal origin. The role of natural killer (NK) cells in pSTS remains poorly understood, with evidence fragmented across small preclinical studies and early‐phase clinical trials.
Raya Dean, Lara V. Graham, Christina Putnam, Rosa Gomes Alves Martins, Daniel T. Harding, Tim J. Underwood, Zoë S. Walters, Matthew D. Blunt   +7 more
wiley   +1 more source

Lentiviral delivery of CRISPR Cas9 prime editing system for cystic fibrosis [PDF]

The field of gene editing is rapidly developing, particularly with advancements in gene editor technologies. Prime editing has been a significant breakthrough in gene editing, allowing precise, targeted correction of all types of DNA mutation, including ...
Satyapertiwi, Dwiantari
core   +1 more source

Recent Advances in Improving Gene-Editing Specificity through CRISPR–Cas9 Nuclease Engineering [PDF]

, 2022
CRISPR–Cas9 is the state-of-the-art programmable genome-editing tool widely used in many areas. For safe therapeutic applications in clinical medicine, its off-target effect must be dramatically minimized.
Yang, D, Zhang, Jifeng, Huang, X, Dongshan Yang, Y. Eugene Chen, Jie Xu, Xiaoqiang Huang, Huang, Xiaoqiang, Jifeng Zhang, Xu, Jie, Xu, J, Yang, Dongshan, Chen, YE, Chen, Y. Eugene, Zhang, J   +14 more
core   +1 more source

Gene Editing for the Treatment of Hypercholesterolemia

Current Atherosclerosis Reports
Abstract Purpose of Review Here, we summarize the key findings from preclinical studies that tested the concept that editing of hepatic genes can lower plasma low-density lipoprotein (LDL)-cholesterol levels to subsequently reduce atherosclerotic cardiovascular disease risk. Recent Findings
Hoekstra, M., Eck, M. van
openaire   +3 more sources

Enteropathogenic E. coli shows delayed attachment and host response in human jejunum organoid‐derived monolayers compared to HeLa cells

FEBS Letters, EarlyView.
Enteropathogenic E. coli (EPEC) infects the human intestinal epithelium, resulting in severe illness and diarrhoea. In this study, we compared the infection of cancer‐derived cell lines with human organoid‐derived models of the small intestine. We observed a delayed in attachment, inflammation and cell death on primary cells, indicating that host ...
Mastura Neyazi, Pilar Samperio Ventayol, Natalie Burkard, Nicolas Schlegel, Carmen Aguilar, Sina Bartfeld   +5 more
wiley   +1 more source

Highly efficient prime editing by introducing same-sense mutations in pegRNA or stabilizing its structure

Nature Communications, 2022
Prime editors can mediate all twelve types of base substitutions and small insertions or deletions in living cells but its efficiency remains low. Here the authors introduce same-sense mutations into pegRNAs to increase base-editing efficiency and the ...
Xiaosa Li, Lina Zhou, Bao-Qing Gao, Guangye Li, Xiao Wang, Ying Wang, Jia Wei, Wenyan Han, Zixian Wang, Jifang Li, Runze Gao, Junjie Zhu, Wenchao Xu, Jing Wu, Bei Yang, Xiaodong Sun, Li Yang, Jia Chen   +17 more
doaj   +1 more source

Reciprocal control of viral infection and phosphoinositide dynamics

FEBS Letters, EarlyView.
Phosphoinositides, although scarce, regulate key cellular processes, including membrane dynamics and signaling. Viruses exploit these lipids to support their entry, replication, assembly, and egress. The central role of phosphoinositides in infection highlights phosphoinositide metabolism as a promising antiviral target.
Marie Déborah Bancilhon, Bruno Mesmin
wiley   +1 more source

Editing-defective MeV are selected in Raji cells. [PDF]

, 2019
Chromatograms of dideoxy-sequencing over the P gene editing site from viruses passaged on Raji or JVM-2 cells. Cell type used and passage numbers are indicated above the chromatograms.
Christian K. Pfaller (6029036), Roberto Cattaneo (363765), Ryan C. Donohue (442093)   +2 more
core   +1 more source