Results 91 to 100 of about 28,868 (258)
Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases
Huntington's disease (HD) is a progressive autosomal dominant neurodegenerative disorder caused by the expansion of CAG repeats in the first exon of the huntingtin gene (HTT).
M. Dabrowska+3 more
semanticscholar +1 more source
Beyond synapses: cytoplasmic connections in brain function and evolution
ABSTRACT Following Ramón y Cajal's groundbreaking contributions to the identification of synapses, research in neuroscience predominantly focused on their pivotal role in neural communication (the neuron doctrine), overlooking an intriguing possibility suggested by Golgi of non‐synaptic interactions among neural cells.
Malalaniaina Rakotobe, Chiara Zurzolo
wiley +1 more source
Huntington’s disease is a severe and currently incurable neurodegenerative disease. An autosomal dominant mutation in the Huntingtin gene (HTT) causes an increase in the polyglutamine fragment length at the protein N-terminus.
Aleksandra S. Taran+3 more
doaj +1 more source
Abnormal brain development in child and adolescent carriers of mutant huntingtin
Objective The huntingtin gene is critical for the formation and differentiation of the CNS, which raises questions about the neurodevelopmental effect of CAG expansion mutations within this gene (mHTT) that cause Huntington disease (HD).
E. van der Plas+7 more
semanticscholar +1 more source
Polymeric nanoparticles represent promising drug (gene) delivery carriers to the brain due to their unique advantages to facilitate the penetration of blood–brain barrier and endocytosis of neuronal cells in neurodegenerative diseases. ABSTRACT In spite of great advances in modern medicine, there are a few effective strategies for the treatment of ...
Lian Jin+4 more
wiley +1 more source
Nuclear Relocation of Normal Huntingtin [PDF]
In Huntington's Disease (HD), the huntingtin protein (Htt) includes an expanded polyglutamine domain. Since mutant Htt concentrates in the nucleus of affected neurons, we have inquired whether normal Htt (Q16−23) is also able to access the nucleus. We observe that a major pool of normal full‐length Htt of HeLa cells is anchored to endosomes and also ...
Tao Tao, Alan M. Tartakoff
openaire +3 more sources
Huntington's disease, with its dominant loss of striatal neurons, is triggered by an expanded glutamine tract in huntingtin. To investigate a proposed role for increased activation of the apoptotic cascade in mutant huntingtin's trigger mechanism, we ...
Shobu Namura+7 more
doaj
Modulation of Huntingtin Toxicity by BAG1 is Dependent on an Intact BAG Domain
Huntington´s disease, one of the so-called poly-glutamine diseases, is a dominantly inherited movement disorder characterized by formation of cytosolic and nuclear inclusion bodies and progressive neurodegeneration.
Mathias Bähr+5 more
doaj +1 more source
Huntington disease (HD) is a fatal neurodegenerative disease caused by a pathogenic expansion of a CAG repeat in the huntingtin (HTT) gene. There are no disease-modifying therapies for HD.
N. Caron+12 more
semanticscholar +1 more source
PI5P4K inhibitors: promising opportunities and challenges
Developing inhibitors is like climbing a steep, challenging mountain. PI5P4K inhibitors hold significant promise for the treatment of cancer, immune disorders, and neurological diseases. However, the path to success is fraught with hurdles, and researchers are therefore required to apply their expertise to overcome critical “checkpoints.” While some ...
Koh Takeuchi+16 more
wiley +1 more source