(Q37045850)

17 August 2017

In vivo gene editing in dystrophic mouse muscle and muscle stem cells (English)

1 reference

17 August 2017

Claire Maesner

7

0 references

Kexian Zhu

object named as

Kexian Zhu

2

0 references

Wei Leong Chew

object named as

Wei Leong Chew

4

0 references

George M. Church

object named as

George M Church

14

0 references

author name string

Mohammadsharif Tabebordbar

1

1 reference

17 August 2017

Jason K W Cheng

3

1 reference

17 August 2017

Jeffrey J Widrick

5

1 reference

17 August 2017

Winston X Yan

6

1 reference

17 August 2017

Elizabeth Y Wu

8

1 reference

17 August 2017

Ru Xiao

9

1 reference

17 August 2017

F Ann Ran

10

1 reference

17 August 2017

Le Cong

11

1 reference

17 August 2017

Feng Zhang

12

1 reference

17 August 2017

Luk H Vandenberghe

13

1 reference

17 August 2017

Amy J Wagers

15

1 reference

17 August 2017

language of work or name

English

0 references

publication date

31 December 2015

1 reference

17 August 2017

1 reference

17 August 2017

351

1 reference

17 August 2017

6271

1 reference

17 August 2017

407-411

1 reference

Clinical features of patients with dystrophinopathy sharing the 45-55 exon deletion of DMD gene

17 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

In vivo genome editing using Staphylococcus aureus Cas9

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Genome engineering using the CRISPR-Cas9 system

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

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

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

RNA-guided human genome engineering via Cas9

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Multiplex genome engineering using CRISPR/Cas systems

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Skeletal muscle degenerative diseases and strategies for therapeutic muscle repair

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

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

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

A robust and high-throughput Cre reporting and characterization system for the whole mouse brain

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Prospective isolation of skeletal muscle stem cells with a Pax7 reporter

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Follow-up of three patients with a large in-frame deletion of exons 45-55 in the Duchenne muscular dystrophy (DMD) gene

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

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

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

A zebrafish embryo culture system defines factors that promote vertebrate myogenesis across species.

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4924477

Elusive sources of variability of dystrophin rescue by exon skipping

4 September 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

DNA targeting specificity of RNA-guided Cas9 nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Isolation of adult mouse myogenic progenitors: functional heterogeneity of cells within and engrafting skeletal muscle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Multiplex Western blotting system for the analysis of muscular dystrophy proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Relationship between muscle fiber types and sizes and muscle architectural properties in the mouse hindlimb

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Direct visualization of the dystrophin network on skeletal muscle fiber membrane

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Association of dystrophin and an integral membrane glycoprotein.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx/mTR mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Engineering adeno-associated viruses for clinical gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Effect of genome size on AAV vector packaging

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Quantitative analysis of the packaging capacity of recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Mechanism of eukaryotic RNA polymerase III transcription termination

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Dynamic imaging of genomic loci in living human cells by an optimized CRISPR/Cas system

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Genome-scale CRISPR-Cas9 knockout screening in human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.AAD5177

Local dystrophin restoration with antisense oligonucleotide PRO051

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26721686

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The molecular basis of muscular dystrophy in the mdx mouse: a point mutation

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26721686

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCIENCE.AAD5177

1 reference

17 August 2017

1 reference

17 August 2017

1 reference