Results 11 to 20 of about 8,339,405 (403)
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
New England Journal of Medicine, 2021 BACKGROUND Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart.
J. Gillmore +25 more
semanticscholar +1 more source
Off-target effects in CRISPR/Cas9 gene editing
Frontiers in Bioengineering and Biotechnology, 2023 Gene editing stands for the methods to precisely make changes to a specific nucleic acid sequence. With the recent development of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system, gene editing has become efficient ...
Congting Guo +3 more
semanticscholar +1 more source
Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
Molecular Cancer, 2022 Clustered regularly interspaced short palindromic repeats (CRISPR) system provides adaptive immunity against plasmids and phages in prokaryotes. This system inspires the development of a powerful genome engineering tool, the CRISPR/CRISPR-associated ...
Si-wei Wang +9 more
semanticscholar +1 more source
Nature Materials, 2021 Endosomal escape remains a fundamental barrier hindering the advancement of nucleic acid therapeutics. Taking inspiration from natural phospholipids that comprise biological membranes, we report the combinatorial synthesis of multi-tailed ionizable ...
Shuai Liu +6 more
semanticscholar +1 more source
Application of CRISPR/Cas9-mediated gene editing for abiotic stress management in crop plants
Frontiers in Plant Science, 2023 Abiotic stresses, including drought, salinity, cold, heat, and heavy metals, extensively reducing global agricultural production. Traditional breeding approaches and transgenic technology have been widely used to mitigate the risks of these environmental
M. Kumar +6 more
semanticscholar +1 more source
Nature Nanotechnology, 2020 CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold tremendous potential to effectively treat disease-causing mutations with diverse cellular origin. However, it is currently impossible to rationally design nanoparticles that
Qiang Cheng +5 more
semanticscholar +1 more source
Engineering the next-generation of CAR T-cells with CRISPR-Cas9 gene editing
Molecular Cancer, 2022 Chimeric Antigen Receptor (CAR) T-cells represent a breakthrough in personalized cancer therapy. In this strategy, synthetic receptors comprised of antigen recognition, signaling, and costimulatory domains are used to reprogram T-cells to target tumor ...
Alexander J Dimitri +2 more
semanticscholar +1 more source
Human Genetics, 2023 Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas) gene-editing system (CRISPR-Cas) is a valuable tool for fundamental and applied research applications.
John Hunt +3 more
semanticscholar +1 more source
RNA Targeting and Gene Editing Strategies for Transthyretin Amyloidosis
BioDrugs, 2023 Transthyretin (TTR) is a tetrameric protein synthesized primarily by the liver. TTR can misfold into pathogenic ATTR amyloid fibrils that deposit in the nerves and heart, causing a progressive and debilitating polyneuropathy (PN) and life-threatening ...
A. Ioannou, M. Fontana, J. Gillmore
semanticscholar +1 more source
Mutations of MSH5 in nonobstructive azoospermia (NOA) and rescued via in vivo gene editing
Signal Transduction and Targeted Therapy, 2022 Dear Editor, Male infertility is a multifactorial heterogeneous pathological condition affecting ∼7% of men. Nonobstructive azoospermia (NOA) is one of the most severe male reproductive diseases and occurs in ∼1% of men of reproductive age.
Min Chen +11 more
semanticscholar +1 more source