[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 …
Precise excision of the CAG tract from the huntingtin gene by Cas9 nickases
M Dabrowska, W Juzwa, WJ Krzyzosiak… - Frontiers in …, 2018 - frontiersin.org
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 …
disorder caused by the expansion of CAG repeats in the first exon of the huntingtin gene …
CRISPR/Cas9 editing of the mutant huntingtin allele in vitro and in vivo
AM Monteys, SA Ebanks, MS Keiser, BL Davidson - Molecular therapy, 2017 - cell.com
Huntington disease (HD) is a fatal dominantly inherited neurodegenerative disorder caused
by CAG repeat expansion (> 36 repeats) within the first exon of the huntingtin gene …
by CAG repeat expansion (> 36 repeats) within the first exon of the huntingtin gene …
Splice modulators target PMS1 to reduce somatic expansion of the Huntington's disease-associated CAG repeat
Huntington's disease (HD) is a dominant neurological disorder caused by an expanded HTT
exon 1 CAG repeat that lengthens huntingtin's polyglutamine tract. Lowering mutant …
exon 1 CAG repeat that lengthens huntingtin's polyglutamine tract. Lowering mutant …
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 …
The potential of gene editing for Huntington's disease
Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by
a trinucleotide repeat expansion in the huntingtin gene resulting in long stretches of …
a trinucleotide repeat expansion in the huntingtin gene resulting in long stretches of …
CRISPR/Cas9 mediated therapeutic approach in Huntington's disease
The pathogenic mechanisms of these diseases must be well understood for the treatment of
neurological disorders such as Huntington's disease. Huntington's Disease (HD), a …
neurological disorders such as Huntington's disease. Huntington's Disease (HD), a …
An RNA-targeting CRISPR–Cas13d system alleviates disease-related phenotypes in Huntington's disease models
KH Morelli, Q Wu, ML Gosztyla, H Liu, M Yao… - Nature …, 2023 - nature.com
Huntington's disease (HD) is a fatal, dominantly inherited neurodegenerative disorder
caused by CAG trinucleotide expansion in exon 1 of the huntingtin (HTT) gene. Since the …
caused by CAG trinucleotide expansion in exon 1 of the huntingtin (HTT) gene. Since the …
Truncation of mutant huntingtin in knock-in mice demonstrates exon1 huntingtin is a key pathogenic form
H Yang, S Yang, L Jing, L Huang, L Chen… - Nature …, 2020 - nature.com
Polyglutamine expansion in proteins can cause selective neurodegeneration, although the
mechanisms are not fully understood. In Huntington's disease (HD), proteolytic processing …
mechanisms are not fully understood. In Huntington's disease (HD), proteolytic processing …
DNA double-strand break-free CRISPR interference delays Huntington's disease progression in mice
JH Seo, JH Shin, J Lee, D Kim, HY Hwang… - Communications …, 2023 - nature.com
Huntington's disease (HD) is caused by a CAG repeat expansion in the huntingtin (HTT)
gene. CRISPR-Cas9 nuclease causes double-strand breaks (DSBs) in the targeted DNA …
gene. CRISPR-Cas9 nuclease causes double-strand breaks (DSBs) in the targeted DNA …