(Q40120224)

English

Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism

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scholarly article

1 reference

Europe PubMed Central

17 September 2017

Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism (English)

1 reference

Europe PubMed Central

17 September 2017

congenital disorder

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congenital muscular dystrophy

1 reference

based on heuristic

inferred from title

congenital merosin-deficient muscular dystrophy 1A

1 reference

based on heuristic

inferred from title

Michael D. Wilson

14

1 reference

Europe PubMed Central

17 September 2017

Dwi U Kemaladewi

object named as

Dwi U Kemaladewi

1

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11

object named as

Daniele Merico

1 reference

Europe PubMed Central

17 September 2017

Brendan J. Frey

13

object named as

Brendan J Frey

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Europe PubMed Central

17 September 2017

author name string

Eleonora Maino

2

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Europe PubMed Central

17 September 2017

Elzbieta Hyatt

3

1 reference

Europe PubMed Central

17 September 2017

Huayun Hou

4

1 reference

Europe PubMed Central

17 September 2017

Maylynn Ding

5

1 reference

Europe PubMed Central

17 September 2017

Kara M Place

6

1 reference

Europe PubMed Central

17 September 2017

Xinyi Zhu

7

1 reference

Europe PubMed Central

17 September 2017

Prabhpreet Bassi

8

1 reference

Europe PubMed Central

17 September 2017

Zahra Baghestani

9

1 reference

Europe PubMed Central

17 September 2017

Amit G Deshwar

10

1 reference

Europe PubMed Central

17 September 2017

Hui Y Xiong

12

1 reference

Europe PubMed Central

17 September 2017

Evgueni A Ivakine

15

1 reference

Europe PubMed Central

17 September 2017

Ronald D Cohn

16

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Europe PubMed Central

17 September 2017

language of work or name

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publication date

17 July 2017

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Europe PubMed Central

17 September 2017

Nature Medicine

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Europe PubMed Central

17 September 2017

23

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Europe PubMed Central

17 September 2017

8

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Europe PubMed Central

17 September 2017

984-989

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Europe PubMed Central

17 September 2017

https://scigraph.springernature.com/pub.10.1038/nm.4367

0 references

RNA mis-splicing in disease

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

More efficient repair of DNA double-strand breaks in skeletal muscle stem cells compared to their committed progeny.

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Laminin-α2 Chain-Deficient Congenital Muscular Dystrophy: Pathophysiology and Development of Treatment

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Identification of a novel mutant transcript of laminin alpha 2 chain gene responsible for muscular dystrophy and dysmyelination in dy2J mice

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

The Pathogenesis and Therapy of Muscular Dystrophies

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

The Human Gene Mutation Database: building a comprehensive mutation repository for clinical and molecular genetics, diagnostic testing and personalized genomic medicine

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Murine muscular dystrophy caused by a mutation in the laminin alpha 2 (Lama2) gene

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

In vivo genome editing using Staphylococcus aureus Cas9

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Pick one, but be quick: 5' splice sites and the problems of too many choices

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Merosin-deficient congenital muscular dystrophy. Partial genetic correction in two mouse models

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Omigapil treatment decreases fibrosis and improves respiratory rate in dy(2J) mouse model of congenital muscular dystrophy

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

In Vivo Assessment of Muscle Contractility in Animal Studies

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Eccentric Contraction-Induced Muscle Injury: Reproducible, Quantitative, Physiological Models to Impair Skeletal Muscle's Capacity to Generate Force

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Structure and Engineering of Francisella novicida Cas9.

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Maximum entropy modeling of short sequence motifs with applications to RNA splicing signals.

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Widespread recognition of 5' splice sites by noncanonical base-pairing to U1 snRNA involving bulged nucleotides

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Noncanonical registers and base pairs in human 5' splice-site selection

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Cell-type specific regulation of myostatin signaling

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Development and applications of CRISPR-Cas9 for genome engineering

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Multiplex genome engineering using CRISPR/Cas systems

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Cas9 as a versatile tool for engineering biology

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

In silico prediction of splice-altering single nucleotide variants in the human genome

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Identification of homozygous and heterozygous dy 2J mice by PCR

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

21 January 2018

Genotype–phenotype correlation in a large population of muscular dystrophy patients with LAMA2 mutations

1 reference

https://api.crossref.org/works/10.1038%2FNM.4367

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1038/NM.4367

1 reference

Europe PubMed Central

17 September 2017

1 reference

Europe PubMed Central

17 September 2017

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