Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy. (Q34463483)

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1 March 2020

title

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

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1 March 2020

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5

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1 March 2020

author name string

David G Ousterout

1

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1 March 2020

Ami M Kabadi

2

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1 March 2020

Pratiksha I Thakore

3

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1 March 2020

William H Majoros

4

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1 March 2020

Charles A Gersbach

6

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language of work or name

English

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publication date

18 February 2015

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1 March 2020

published in

Nature Communications

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1 March 2020

volume

6

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1 March 2020

page(s)

6244

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https://scigraph.springernature.com/pub.10.1038/ncomms7244

1 March 2020

exact match

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cites work

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6 July 2018

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6 July 2018

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6 July 2018

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Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in.

6 July 2018

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Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.

6 July 2018

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6 July 2018

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6 July 2018

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6 July 2018

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6 July 2018

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

6 July 2018

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6 July 2018

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6 July 2018

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Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation

6 July 2018

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6 July 2018

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6 July 2018

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TALENs: a widely applicable technology for targeted genome editing

6 July 2018

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

6 July 2018

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Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice.

6 July 2018

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

6 July 2018

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6 July 2018

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6 July 2018

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6 July 2018

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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

6 July 2018

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6 July 2018

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6 July 2018

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Systemic administration of PRO051 in Duchenne's muscular dystrophy

6 July 2018

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Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice

6 July 2018

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6 July 2018

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The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy

6 July 2018

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Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells

6 July 2018

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6 July 2018

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Chromosomal translocations induced at specified loci in human stem cells.

6 July 2018

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Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

6 July 2018

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6 July 2018

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Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

6 July 2018

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6 July 2018

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6 July 2018

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Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

6 July 2018

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Dystrophin: the protein product of the Duchenne muscular dystrophy locus

6 July 2018

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Microhomology-based choice of Cas9 nuclease target sites.

6 July 2018

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Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

21 January 2018

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

21 January 2018

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Welcome to skeletal muscle

21 January 2018

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Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25692716

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/NCOMMS7244

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25692716%20AND%20SRC:MED&resulttype=core&format=json

Dimensions Publication ID

1 March 2020

ADS bibcode

2015NatCo...6.6244O

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0 references

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25692716%20AND%20SRC:MED&resulttype=core&format=json

1 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25692716%20AND%20SRC:MED&resulttype=core&format=json