CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy (Q38919946)

7 September 2017

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy (English)

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7 September 2017

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1 reference

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Thierry VandenDriessche

series ordinal

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

1 June 2020

Marinee K Chuah

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

1 June 2020

publication date

1 March 2016

1 reference

7 September 2017

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

1 June 2020

volume

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

1 June 2020

issue

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

1 June 2020

page(s)

414-416

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method.

1 June 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations.

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function 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=4786932

Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD).

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses.

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

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

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

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

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

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=4786932

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

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

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=4786932

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=4786932

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=4786932

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

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic 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=4786932

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy

9 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Association of dystrophin and an integral membrane glycoprotein.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo

22 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4786932

Local dystrophin restoration with antisense oligonucleotide PRO051

22 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26952918

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26952918

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2016.29

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

1 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json

1 June 2020

PubMed publication ID

26952918

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26952918%20AND%20SRC:MED&resulttype=core&format=json