Results 31 to 40 of about 869,701 (314)

Gene editing for inflammatory disorders [PDF]

open access: yesAnnals of the Rheumatic Diseases, 2019
Technology for precise and efficient genetic editing is constantly evolving and is now capable of human clinical applications. Autoimmune and inflammatory diseases are chronic, disabling, sometimes life-threatening, conditions that feature heritable components.
David T, Ewart   +2 more
openaire   +2 more sources

Strategies for mitochondrial gene editing

open access: yesComputational and Structural Biotechnology Journal, 2021
Mitochondria, as the energy factory of cells, participate in metabolism processes and play a critical role in the maintenance of human life activities. Mitochondria belong to semi-automatic organelles, which have their own genome different from nuclear genome. Mitochondrial DNA (mtDNA) mutations can cause a series of diseases and threaten human health.
Xingbo Yang   +4 more
openaire   +3 more sources

Delivery of CRISPR/Cas9 Plasmid DNA by Hyperbranched Polymeric Nanoparticles Enables Efficient Gene Editing [PDF]

open access: yes, 2022
Gene editing nucleases such as CRISPR/Cas9 have enabled efficient and precise gene editing in vitro and hold promise of eventually achieving in vivo gene editing based therapy.
Luan Wen   +10 more
core   +1 more source

Endogenous leptin promotes autophagy in EBSS-induced PFCs

open access: yesAnimal Cells and Systems, 2019
Leptin is an important adipokine and plays a vital role in animals. However, the role of leptin in the autophagic response of pig fibroblast cells (PFCs) has not been fully elucidated.
Deling Jiao   +10 more
doaj   +1 more source

CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives [PDF]

open access: yes, 2022
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-of-function mutations in the dystrophin gene and characterized by progressive muscle degeneration, respiratory insufficiency, cardiac failure, and premature death by ...
Hui Yang   +9 more
core   +1 more source

Gene Editing

open access: yes, 2023
Gene editing allows scientists to modify an organism's DNA with unparalleled precision, utilizing techniques like CRISPR-Cas9. It aids researchers to target specific genes, enhance desirable traits, correct genetic disorders, and develop innovative therapies.
openaire   +3 more sources

Cell Surface-Expressed GPI-Anchored Peptides from the CHR Domain of gp41 Are Potent Inhibitors of HIV-1 Fusion

open access: yesProceedings, 2020
Current antiretroviral therapy efficiently suppresses viral replication but cannot eliminate latent HIV reservoirs. Moreover, the associated high costs, side effects, and drug resistance have stimulated a need for the development of alternative methods ...
Aleksandra Maslennikova   +3 more
doaj   +1 more source

CRISPR Gene Editing in the Kidney [PDF]

open access: yesAmerican Journal of Kidney Diseases, 2018
CRISPR is a nuclease guidance system that enables rapid and efficient gene editing of specific DNA sequences within genomes. We review applications of CRISPR for the study and treatment of kidney disease. CRISPR enables functional experiments in cell lines and model organisms to validate candidate genes arising from genetic studies.
Nelly M, Cruz, Benjamin S, Freedman
openaire   +2 more sources

Gene Editing: Medicine or enhancement [PDF]

open access: yes, 2020
In this paper we will discuss the status of gene editing technologies like CRISPR. We will examine whether this technology should be considered a form of enhancement, or if CRISPR is merely a medical technology analogous to many of the common medical ...
Anomaly, Jonathan   +2 more
core  

CRISPR/dCas9-Dnmt3a-mediated targeted DNA methylation of APP rescues brain pathology in a mouse model of Alzheimer’s disease

open access: yesTranslational Neurodegeneration, 2022
Background Aberrant DNA methylation patterns have been observed in neurodegenerative diseases, including Alzheimer's disease (AD), and dynamic changes in DNA methylation are closely associated with the onset and progression of these diseases ...
Hanseul Park   +5 more
doaj   +1 more source

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