Results 61 to 70 of about 8,657,992 (314)

CRISPR–Cas9 Gene Editing: Curing Genetic Diseases by Inherited Epigenetic Modifications

Global Medical Genetics
Introduction  CRISPR–Cas9 gene editing, leveraging bacterial defense mechanisms, offers precise DNA modifications, holding promise in curing genetic diseases.
Nikhil Deep Kolanu
semanticscholar   +1 more source

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.

New England Journal of Medicine, 2020
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin in
H. Frangoul, D. Altshuler, M. Cappellini, Yi-Shan Chen, J. Domm, Brenda K. Eustace, J. Foell, J. de la Fuente, S. Grupp, R. Handgretinger, Tony Ho, A. Kattamis, A. Kernytsky, J. Lekstrom-Himes, Amanda M. Li, F. Locatelli, M. Mapara, M. de Montalembert, D. Rondelli, Akshay Sharma, S. Sheth, S. Soni, M. Steinberg, D. Wall, Angela Yen, S. Corbacioglu   +25 more
semanticscholar   +1 more source

Advances in Delivery Mechanisms of CRISPR Gene-Editing Reagents in Plants

Frontiers in Genome Editing, 2022
Gene-editing by CRISPR/Cas systems has revolutionized plant biology by serving as a functional genomics tool. It has tremendously advanced plant breeding and crop improvement by accelerating the development of improved cultivars, creating genetic ...
Larissa Laforest, S. S. Nadakuduti
semanticscholar   +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

A global observatory for gene editing [PDF]

Nature, 2018
Sheila Jasanoff and J. Benjamin Hurlbut call for an international network of scholars and organizations to support a new kind of conversation. Sheila Jasanoff and J. Benjamin Hurlbut call for an international network of scholars and organizations to support a new kind of conversation.
Sheila, Jasanoff, J Benjamin, Hurlbut
openaire   +2 more sources

CRISPR-Cas9-mediated homology-directed repair for precise gene editing

Molecular Therapy: Nucleic Acids
CRISPR-Cas9-mediated homology-directed repair (HDR) is a versatile platform for creating precise site-specific DNA insertions, deletions, and substitutions. These precise edits are made possible through the use of exogenous donor templates that carry the
Hongyu Liao, Jiahao Wu, Nathan J. VanDusen, Yifei Li, Yanjiang Zheng   +4 more
semanticscholar   +1 more source

Spatiotemporal control of CRISPR/Cas9 gene editing

Signal Transduction and Targeted Therapy, 2021
The clustered regularly interspaced short palindromic repeats (CRISPR)/associated protein 9 (CRISPR/Cas9) gene editing technology, as a revolutionary breakthrough in genetic engineering, offers a promising platform to improve the treatment of various ...
Chenya Zhuo, Jiabin Zhang, Jung-Hwan Lee, J. Jiao, Du Cheng, Li Liu, H. Kim, Yu Tao, Mingqiang Li   +8 more
semanticscholar   +1 more source

Phosphatidylinositol 4‐kinase as a target of pathogens—friend or foe?

FEBS Letters, EarlyView.
This graphical summary illustrates the roles of phosphatidylinositol 4‐kinases (PI4Ks). PI4Ks regulate key cellular processes and can be hijacked by pathogens, such as viruses, bacteria and parasites, to support their intracellular replication. Their dual role as essential host enzymes and pathogen cofactors makes them promising drug targets.
Ana C. Mendes, Guilherme M. Azevedo, Amanda P. Barcellos, Diana Bahia   +3 more
wiley   +1 more source

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

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