(Q48041681)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

title

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

Collaborative Research Centre (CRC) 1002: "Modulatory Units in Heart Failure"

14 March 2020

sponsor

0 references

German Research Foundation

0 references

1 reference

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Hui Li

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

Cristina Rodríguez-Caycedo

14 March 2020

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Huanyu Zhou

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Yu Zhang

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Yi-Li Min

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

John M Shelton

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Pradeep P A Mammen

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Norman Yu Liaw

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

Wolfram-Hubertus Zimmermann

14 March 2020

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Rhonda Bassel-Duby

13

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Eric N. Olson

15

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

author name string

Malte Tiburcy

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Viktoriia Kyrychenko

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

Jay W Schneider

14

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

publication date

31 January 2018

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

volume

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

issue

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

page(s)

eaap9004

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

Functional correction of dystrophin actin binding domain mutations by genome editing

14 March 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

The importance of genetic diagnosis for Duchenne muscular dystrophy

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Rationally engineered Cas9 nucleases with improved specificity

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

DNA targeting specificity of RNA-guided Cas9 nucleases

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

RNA-guided human genome engineering via Cas9

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Multiplex genome engineering using CRISPR/Cas systems

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Overview on DMD exon skipping

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Double-strand break end resection and repair pathway choice

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Cardiomyopathy in Duchenne muscular dystrophy: pathogenesis and therapeutics

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

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

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.

22 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Correction of a pathogenic gene mutation in human embryos

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Defined Engineered Human Myocardium With Advanced Maturation for Applications in Heart Failure Modeling and Repair.

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects.

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Distinct metabolic flow enables large-scale purification of mouse and human pluripotent stem cell-derived cardiomyocytes

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Prevention of dystrophin-deficient cardiomyopathy in twenty-one-month-old carrier mice by mosaic dystrophin expression or complementary dystrophin/utrophin expression

14 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5796795

Dp140: a novel 140 kDa CNS transcript from the dystrophin locus

14 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29404407

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

DMD pseudoexon mutations: splicing efficiency, phenotype, and potential therapy

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29404407

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Evidence-based path to newborn screening for Duchenne muscular dystrophy

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/29404407

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCIADV.AAP9004

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29404407%20AND%20SRC:MED&resulttype=core&format=json

14 March 2020

PubMed publication ID

29404407

1 reference