Results 11 to 20 of about 8,657,992 (314)
Gene editing therapeutics based on mRNA delivery
Advanced Drug Delivery Reviews, 2023 The field of gene editing has received much attention in recent years due to its immense therapeutic potential. In particular, gene editing therapeutics, such as the CRISPR-Cas systems, base editors, and other emerging gene editors, offer the opportunity
Beob Soo Kim, Niren Murthy, Kunwoo Lee
exaly +2 more sources
Gene Editing, Identity and Benefit [PDF]
The Philosophical Quarterly, 2021 Abstract Some suggest that gene editing human embryos to prevent genetic disorders will be in one respect morally preferable to using genetic selection for the same purpose: gene editing will benefit particular future persons, while genetic selection would merely replace them.
Douglas, T, Devolder, K
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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
Life, 2021 The base editing 3 (BE3) system, a single-base gene editing technology developed using CRISPR/Cas9n, has a broad range of applications for human disease model construction and gene therapy, as it is highly efficient, accurate, and non-destructive.
Honghui Li +13 more
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Homology directed correction, a new pathway model for point mutation repair catalyzed by CRISPR-Cas
Scientific Reports, 2022 Gene correction is often referred to as the gold standard for precise gene editing and while CRISPR-Cas systems continue to expand the toolbox for clinically relevant genetic repair, mechanistic hurdles still hinder widespread implementation.
Brett M. Sansbury +5 more
doaj +1 more source
Construction of PIK3C3 Transgenic Pig and Its Pathogenesis of Liver Damage
Life, 2022 As a member of the PIKs family, PIK3C3 participates in autophagy and plays a central role in liver function. Several studies demonstrated that the complete suppression of PIK3C3 in mammals can cause hepatomegaly and hepatosteatosis. However, the function
Jing Wang +19 more
doaj +1 more source
Gene editing and gene regulation with CRISPR [PDF]
Experimental Physiology, 2018 Genome editing enables precise changes to be made in the genome of living cells. The technique was originally developed in the 1980’s but largely limited to use in mice. The discovery that a targeted double stranded break (DSB) at a unique site in the genome, close to the site to be changed, could substantially increase the efficiency of editing ...
Patrick Harrison, Stephen Hart
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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
The origin of unwanted editing byproducts in gene editing
Acta Biochimica et Biophysica Sinica, 2022 The rapid development of CRISPR-Cas genome editing tools has greatly changed the way to conduct research and holds tremendous promise for clinical applications. During genome editing, CRISPR-Cas enzymes induce DNA breaks at the target sites and subsequently the DNA repair pathways are recruited to generate diverse editing outcomes.
Yin Jianhang, Hu Jiazhi
openaire +4 more sources
Establishment of PCR Identification Method for Pig Blood Type
Shiyan dongwu yu bijiao yixue, 2023 ObjectiveXenotransplantation is an effective way to address the shortage of human organ donors, but it faces serious immune rejection reactions, including hyperacute rejection caused by blood type differences.
WANG Jiaoxiang +8 more
doaj +1 more source