Results 51 to 60 of about 571,422 (359)
Switchable genome editing via genetic code expansion [PDF]
, 2018 Multiple applications of genome editing by CRISPR-Cas9 necessitate stringent regulation and Cas9 variants have accordingly been generated whose activity responds to small ligands, temperature or light.
Asami, Maki +5 more
core +2 more sources
Genome edited sheep and cattle [PDF]
Transgenic Research, 2014 Genome editing tools enable efficient and accurate genome manipulation. An enhanced ability to modify the genomes of livestock species could be utilized to improve disease resistance, productivity or breeding capability as well as the generation of new biomedical models. To date, with respect to the direct injection of genome editor mRNA into livestock
Proudfoot, Christopher +10 more
openaire +4 more sources
Genome Editing for CNS Disorders [PDF]
Frontiers in Neuroscience, 2020 Central nervous system (CNS) disorders have a social and economic burden on modern societies, and the development of effective therapies is urgently required. Gene editing may prevent or cure a disease by inducing genetic changes at endogenous loci. Genome editing includes not only the insertion, deletion or replacement of nucleotides, but also the ...
Fábio Duarte +3 more
openaire +5 more sources
Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications [PDF]
, 2017 BACKGROUND: Recently established genome editing technologies will open new avenues for biological research and development. Human genome editing is a powerful tool which offers great scientific and therapeutic potential.CONTENT: Genome editing using the ...
Dewi, N. M. (Nurrani) +2 more
core +4 more sources
Large-Scale CRISPR Screen of LDLR Pathogenic Variants
Research, 2023 Familial hypercholesterolemia (FH) is a frequently occurring genetic disorder that is linked to early-onset cardiovascular disease. If left untreated, patients with this condition can develop severe cardiovascular complications.
Mengjing Li +9 more
doaj +1 more source
Breakthrough to genome editing [PDF]
Science, 2015 A, T, G, C: the alphabet code for the nucleotides that are the building blocks of life. Minor, but consequential, changes in this DNA coding can change gene function. Researchers have long sought better ways to edit the genetic code in cultured cells and laboratory organisms to silence, activate, or change targeted genes to gain a better ...
openaire +3 more sources
Editing the Mitochondrial Genome
New England Journal of Medicine, 2020 Mending Mitochondrial Mutation For most patients with mitochondrial disorders, most of which are caused by mutations in genes in the mitochondrial genome, only symptomatic treatment is available.
Maria Falkenberg, Michio Hirano
openaire +3 more sources
Comprehensive assessment of miniature CRISPR-Cas12f nucleases for gene disruption
Nature Communications, 2022 CRISPR-Cas12f nucleases can be effectively packaged into AAVs for gene therapy, but a systematic evaluation of editing outcomes is lacking. Here the authors perform a comprehensive assessment of 4 Cas12f proteins and compare to Cas9 and two Cas12a ...
Changchang Xin +6 more
doaj +1 more source
Frontiers in Immunology, 2023 Regulatory B lymphocytes (Bregs) are B cells with well-pronounced immunosuppressive properties, allowing them to suppress the activity of effector cells.
Elina A. Zheremyan +11 more
doaj +1 more source
Improving single-cell cloning workflow for gene editing in human pluripotent stem cells [PDF]
, 2018 The availability of human pluripotent stem cells (hPSCs) and progress in genome engineering technology have altered the way we approach scientific research and drug development screens.
Chen, Yi-Hsien, Pruett-Miller, Shondra M
core +2 more sources