Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders (Q34045672)

30 July 2017

title

Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders (English)

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30 July 2017

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Dwi U Kemaladewi

2

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Michael D. Wilson

object named as

Michael D Wilson

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Sergio L Pereira

object named as

Sergio Pereira

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Vincent Mouly

object named as

Vincent Mouly

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author name string

Daria Wojtal

1

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30 July 2017

Zeenat Malam

3

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30 July 2017

Sarah Abdullah

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Tatianna W Y Wong

5

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Elzbieta Hyatt

6

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Zahra Baghestani

7

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James Stavropoulos

9

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Kamel Mamchaoui

11

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Francesco Muntoni

12

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Thomas Voit

13

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Hernan D Gonorazky

14

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James J Dowling

15

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Roberto Mendoza-Londono

17

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Evgueni A Ivakine

18

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Ronald D Cohn

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30 July 2017

language of work or name

English

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publication date

9 December 2015

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American Journal of Human Genetics

30 July 2017

published in

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30 July 2017

volume

98

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30 July 2017

issue

1

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30 July 2017

page(s)

90-101

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CRISPRscan: designing highly efficient sgRNAs for CRISPR-Cas9 targeting in vivo

30 July 2017

cites work

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5 July 2018

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Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

5 July 2018

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Integrative analysis of 111 reference human epigenomes

5 July 2018

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Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

5 July 2018

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GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

5 July 2018

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5 July 2018

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5 July 2018

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Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.

5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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Characterization of genomic deletion efficiency mediated by clustered regularly interspaced palindromic repeats (CRISPR)/Cas9 nuclease system in mammalian cells.

5 July 2018

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Development and applications of CRISPR-Cas9 for genome engineering

5 July 2018

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Efficient mutagenesis by Cas9 protein-mediated oligonucleotide insertion and large-scale assessment of single-guide RNAs

5 July 2018

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A promoter-level mammalian expression atlas

5 July 2018

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Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

5 July 2018

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Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients

5 July 2018

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New developments in the use of gene therapy to treat Duchenne muscular dystrophy

5 July 2018

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Genome engineering using the CRISPR-Cas9 system

5 July 2018

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Laminin-111 improves muscle repair in a mouse model of merosin-deficient congenital muscular dystrophy

5 July 2018

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Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system

5 July 2018

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CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

5 July 2018

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CRISPR RNA-guided activation of endogenous human genes

5 July 2018

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DNA targeting specificity of RNA-guided Cas9 nucleases

5 July 2018

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CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes

5 July 2018

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Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice

5 July 2018

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5 July 2018

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RNA-guided human genome engineering via Cas9

5 July 2018

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Multiplex genome engineering using CRISPR/Cas systems

5 July 2018

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An expansive human regulatory lexicon encoded in transcription factor footprints

5 July 2018

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5 July 2018

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Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

5 July 2018

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5 July 2018

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The effects of low levels of dystrophin on mouse muscle function and pathology

5 July 2018

1 reference

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5 July 2018

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Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders

5 July 2018

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Genome-wide translocation sequencing reveals mechanisms of chromosome breaks and rearrangements in B cells

5 July 2018

1 reference

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Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma

5 July 2018

1 reference

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

5 July 2018

1 reference

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Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4716669

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

5 July 2018

1 reference

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Systemic administration of PRO051 in Duchenne's muscular dystrophy

5 July 2018

1 reference

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Targeted chromosomal deletions in human cells using zinc finger nucleases

5 July 2018

1 reference

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Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression.

5 July 2018

1 reference

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Short motif sequences determine the targets of the prokaryotic CRISPR defence system

5 July 2018

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CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes

5 July 2018

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Global variation in copy number in the human genome

5 July 2018

1 reference

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Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin

5 July 2018

1 reference

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Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements

5 July 2018

1 reference

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CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies.

5 July 2018

1 reference

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Exploring strategies for the design of artificial transcription factors: targeting sites proximal to known regulatory regions for the induction of gamma-globin expression and the treatment of sickle cell disease

5 July 2018

1 reference

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Gene Length and Proximity to Neighbors Affect Genome-Wide Expression Levels

5 July 2018

1 reference

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Positive and negative regulation of endogenous genes by designed transcription factors

5 July 2018

1 reference

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A second promoter provides an alternative target for therapeutic up-regulation of utrophin in Duchenne muscular dystrophy

5 July 2018

1 reference

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Expression of full-length utrophin prevents muscular dystrophy in mdx mice

5 July 2018

1 reference

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Molecular and functional analysis of the utrophin promoter

5 July 2018

1 reference

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Integrated study of 100 patients with Xp21 linked muscular dystrophy using clinical, genetic, immunochemical, and histopathological data. Part 1. Trends across the clinical groups

5 July 2018

1 reference

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Dystrophin expression in Duchenne patients with "in-frame" gene deletions.

5 July 2018

1 reference

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Low dystrophin levels in heart can delay heart failure in mdx mice.

5 July 2018

1 reference

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Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

28 October 2018

1 reference

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Synergistic and tunable human gene activation by combinations of synthetic transcription factors

28 October 2018

1 reference

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Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4716669

Sequential recruitment of HAT and SWI/SNF components to condensed chromatin by VP16.

28 October 2018

1 reference

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Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands

28 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26686765

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1016/J.AJHG.2015.11.012

1 reference

30 July 2017

1 reference

30 July 2017

1 reference