The biology of huntingtin
F Saudou, S Humbert - Neuron, 2016 - cell.com
Huntingtin (HTT) is now a famous protein because an abnormal expansion of a glutamine
stretch (polyQ) in its N-terminal sequence leads to the devastating neurodegenerative …
stretch (polyQ) in its N-terminal sequence leads to the devastating neurodegenerative …
New avenues for the treatment of Huntington's disease
A Kim, K Lalonde, A Truesdell, P Gomes Welter… - International journal of …, 2021 - mdpi.com
Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG expansion in
the HD gene. The disease is characterized by neurodegeneration, particularly in the striatum …
the HD gene. The disease is characterized by neurodegeneration, particularly in the striatum …
[HTML][HTML] CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease
Huntington's disease is a neurodegenerative disorder caused by a polyglutamine repeat in
the Huntingtin gene (HTT). Although suppressing the expression of mutant HTT (mHTT) has …
the Huntingtin gene (HTT). Although suppressing the expression of mutant HTT (mHTT) has …
CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington's disease mouse model
FK Ekman, DS Ojala, MM Adil, PA Lopez… - … Therapy-Nucleic Acids, 2019 - cell.com
Huntington's disease (HD) is a currently incurable and, ultimately, fatal neurodegenerative
disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin …
disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin …
Molecular strategies to target protein aggregation in Huntington's disease
OD Jarosińska, SGD Rüdiger - Frontiers in molecular biosciences, 2021 - frontiersin.org
Huntington's disease (HD) is a neurodegenerative disorder caused by the aggregation of
the mutant huntingtin (mHTT) protein in nerve cells. mHTT self-aggregates to form soluble …
the mutant huntingtin (mHTT) protein in nerve cells. mHTT self-aggregates to form soluble …
Design, characterization, and lead selection of therapeutic miRNAs targeting huntingtin for development of gene therapy for Huntington's disease
J Miniarikova, I Zanella, A Huseinovic… - … Therapy-Nucleic Acids, 2016 - cell.com
Huntington's disease (HD) is a neurodegenerative disorder caused by accumulation of CAG
expansions in the huntingtin (HTT) gene. Hence, decreasing the expression of mutated HTT …
expansions in the huntingtin (HTT) gene. Hence, decreasing the expression of mutated HTT …
The self-inactivating KamiCas9 system for the editing of CNS disease genes
N Merienne, G Vachey, L de Longprez, C Meunier… - Cell reports, 2017 - cell.com
Neurodegenerative disorders are a major public health problem because of the high
frequency of these diseases. Genome editing with the CRISPR/Cas9 system is making it …
frequency of these diseases. Genome editing with the CRISPR/Cas9 system is making it …
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models
Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and
sustained silencing of gene expression in vivo. We recently demonstrated that siRNA …
sustained silencing of gene expression in vivo. We recently demonstrated that siRNA …
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease
J Miniarikova, V Zimmer, R Martier, CC Brouwers… - Gene therapy, 2017 - nature.com
Huntington's disease (HD) is a fatal progressive neurodegenerative disorder caused by a
mutation in the huntingtin (HTT) gene. To date, there is no treatment to halt or reverse the …
mutation in the huntingtin (HTT) gene. To date, there is no treatment to halt or reverse the …
Huntington disease as a neurodevelopmental disorder and early signs of the disease in stem cells
K Wiatr, WJ Szlachcic, M Trzeciak, M Figlerowicz… - Molecular …, 2018 - Springer
Huntington disease (HD) is a dominantly inherited disorder caused by a CAG expansion
mutation in the huntingtin (HTT) gene, which results in the HTT protein that contains an …
mutation in the huntingtin (HTT) gene, which results in the HTT protein that contains an …