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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.
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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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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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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
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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
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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
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Small Ruminant Farming in Tribal Areas of Dera Ghazi Khan, Punjab, Pakistan
Veterinary Sciences, 2022 Provincially Administered Tribal Areas (PATA) of Punjab-Pakistan are comprised of hilly mountains with small ruminants as a sole source of income. In this study, farming practices, productivity, health and the economic value of sheep were evaluated in ...
Muhammad Ameen Jamal +12 more
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The Ethics of Germline Gene Editing [PDF]
Journal of Applied Philosophy, 2016 AbstractGermline Gene Editing (GGE) has enormous potential both as a research tool and a therapeutic intervention. While other types of gene editing are relatively uncontroversial, GGE has been strongly resisted. In this article, we analyse the ethical arguments for and against pursuing GGE by allowing and funding its development.
Gyngell, Christopher +2 more
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