Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9. (Q48840079)

16 February 2018

title

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9. (English)

1 reference

16 February 2018

0 references

0 references

1 reference

1 reference

James F Gusella

1 reference

16 February 2018

Tammy Gillis

Tammy Gillis

1 reference

16 February 2018

Marcy E. MacDonald

Marcy E MacDonald

1 reference

16 February 2018

Ranjit S Atwal

Ranjit S Atwal

1 reference

16 February 2018

Jun Wan Shin

Jun Wan Shin

1 reference

16 February 2018

author name string

Kyung-Hee Kim

1 reference

16 February 2018

Michael J Chao

1 reference

16 February 2018

Jong-Min Lee

1 reference

16 February 2018

publication date

1 October 2016

1 reference

16 February 2018

published in

Human Molecular Genetics

1 reference

16 February 2018

volume

1 reference

16 February 2018

issue

1 reference

16 February 2018

page(s)

4566-4576

1 reference

16 February 2018

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes.

cites work

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Trinucleotide Repeat Disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Common SNP-based haplotype analysis of the 4p16.3 Huntington disease gene region

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

The relationship between trinucleotide (CAG) repeat length and clinical features of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Trinucleotide repeat length instability and age of onset in Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Relationship between trinucleotide repeat expansion and phenotypic variation in Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

The HTT CAG-Expansion Mutation Determines Age at Death but Not Disease Duration in Huntington Disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Identification of Genetic Factors that Modify Clinical Onset of Huntington's Disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Cleavage pattern and emerging asymmetry of the mouse embryo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Huntington's disease: from molecular pathogenesis to clinical treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Silencing polyglutamine degeneration with RNAi

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Antisense oligonucleotide therapy for neurodegenerative disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Sequence-Level Analysis of the Major European Huntington Disease Haplotype

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Structure and expression of the Huntington's disease gene: evidence against simple inactivation due to an expanded CAG repeat

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Development and applications of CRISPR-Cas9 for genome engineering

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

DNA targeting specificity of RNA-guided Cas9 nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Multi-kilobase homozygous targeted gene replacement in human induced pluripotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

HD iPSC-derived neural progenitors accumulate in culture and are susceptible to BDNF withdrawal due to glutamate toxicity

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Inactivation of the mouse Huntington's disease gene homolog Hdh

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Efficient and allele-specific genome editing of disease loci in human iPSCs

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Rationally engineered Cas9 nucleases with improved specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR-Cas platform

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

CRISPR/Cas9 DNA cleavage at SNP-derived PAM enables both in vitro and in vivo KRT12 mutation-specific targeting

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Induced pluripotent stem cell lines derived from human somatic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDW286

Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia

21 January 2018

1 reference

PubMed Central

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6078600

Normal huntingtin function: an alternative approach to Huntington's disease

2 November 2018

1 reference

PubMed Central

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6078600

2 November 2018

Identifiers

DOI

10.1093/HMG/DDW286

1 reference

16 February 2018

PMC publication ID

6078600

0 references

1 reference

16 February 2018