Results 1 to 10 of about 7,473,287 (361)
All‐In‐One Dendrimer‐Based Lipid Nanoparticles Enable Precise HDR‐Mediated Gene Editing In Vivo
Advanced Materials, 2021 Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR‐associated (Cas) protein gene editing is poised to transform the treatment of genetic diseases.
Daniel J Siegwart +2 more
exaly +2 more sources
Therapeutic Gene Editing for Hemoglobinopathies. [PDF]
Mediterr J Hematol Infect DisIn the last ten years, a consistent number of clinical studies have evaluated different gene approaches for the treatment of patients with sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Initial studies of gene therapy for hemoglobinopathies involved the use of lentiviral vectors to add functional copies of the gene encoding β ...
Testa U, Leone G, Cappellini MD.
europepmc +4 more sources
Therapeutic Gene Editing in Dyslipidemias. [PDF]
Rev Cardiovasc MedDyslipidemia, characterized by abnormal lipid levels in the blood, significantly escalates the risk of atherosclerotic cardiovascular disease and requires effective treatment strategies. While existing therapies can be effective, long-term adherence is often challenging. There has been an interest in developing enduring and more efficient solutions. In
Tamehri Zadeh SS, Shapiro MD.
europepmc +4 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
openaire +3 more sources
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
doaj +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
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
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
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