Results 1 to 10 of about 120,353 (299)

Aberrant splicing of mutant huntingtin in Huntington's disease knock-in pigs

open access: yesNeurobiology of Disease, 2023
Huntington's disease (HD) is an autosomal-dominant inherited neurodegenerative disease caused by a CAG repeat expansion in exon1 of the huntingtin gene (HTT).
Huichun Tong   +12 more
doaj   +1 more source

Intravenous AAV9 administration results in safe and widespread distribution of transgene in the brain of mini-pig

open access: yesFrontiers in Cell and Developmental Biology, 2023
Animal models are important for understanding the pathogenesis of human diseases and for developing and testing new drugs. Pigs have been widely used in the research on the cardiovascular, skin barrier, gastrointestinal, and central nervous systems as ...
Yingqi Lin   +16 more
doaj   +1 more source

Generation of inactivated IL2RG and RAG1 monkeys with severe combined immunodeficiency using base editing

open access: yesSignal Transduction and Targeted Therapy, 2023
Severe combined immunodeficiency (SCID) encompasses a range of inherited disorders that lead to a profound deterioration of the immune system. Among the pivotal genes associated with SCID, RAG1 and IL2RG play crucial roles.
Xiao Zheng   +17 more
doaj   +1 more source

Dynamic Regulation of DNA Methylation and Brain Functions

open access: yesBiology, 2023
DNA cytosine methylation is a principal epigenetic mechanism underlying transcription during development and aging. Growing evidence suggests that DNA methylation plays a critical role in brain function, including neurogenesis, neuronal differentiation ...
Jiaxiang Xie   +4 more
doaj   +1 more source

Insights into White Matter Defect in Huntington’s Disease

open access: yesCells, 2022
Huntington’s disease (HD) is an autosomal-dominant inherited progressive neurodegenerative disorder. It is caused by a CAG repeat expansion in the Huntingtin gene that is translated to an expanded polyglutamine (PolyQ) repeat in huntingtin protein. HD is
Yize Sun   +5 more
doaj   +1 more source

Microbiome Studies in Non-human Primates [PDF]

open access: yesCurrent HIV/AIDS Reports, 2021
Observations of differing bacterial, intestinal microbiomes in people living with HIV have propelled interest in contributions of the microbiome to HIV disease. Non-human primate (NHP) models of HIV infection provide a controlled setting for assessing contributions of the microbiome by standardizing environmental confounders.
Brenchley, Jason M., Ortiz, Alexandra M.
openaire   +2 more sources

A new non-human primate model of desiccating stress-induced dry eye disease

open access: yesScientific Reports, 2022
Dry eye disease (DED), a multifactorial ocular surface disease, is estimated to affect up to 34% of individuals over 50 years old. Although numerous animal models, including rodents and rabbits, have been developed to mimic the pathophysiologic ...
Li Gong   +9 more
doaj   +1 more source

Genetically modified large animal models for investigating neurodegenerative diseases

open access: yesCell & Bioscience, 2021
Neurodegenerative diseases represent a large group of neurological disorders including Alzheimer’s disease, amyotrophic lateral sclerosis, Parkinson’s disease, and Huntington’s disease.
Weili Yang   +3 more
doaj   +1 more source

High-titer AAV disrupts cerebrovascular integrity and induces lymphocyte infiltration in adult mouse brain

open access: yesMolecular Therapy: Methods & Clinical Development, 2023
The brain is often described as an “immune-privileged” organ due to the presence of the blood-brain-barrier (BBB), which limits the entry of immune cells.
Yaowei Guo   +10 more
doaj   +1 more source

A Specific Mini‐Intrabody Mediates Lysosome Degradation of Mutant Huntingtin

open access: yesAdvanced Science, 2023
Accumulation of misfolded proteins leads to many neurodegenerative diseases that can be treated by lowering or removing mutant proteins. Huntington's disease (HD) is characterized by the intracellular accumulation of mutant huntingtin (mHTT) that can be ...
Caijuan Li   +16 more
doaj   +1 more source

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