CRISPR Correction of Duchenne Muscular Dystrophy (Q58086427)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

1 May 2020

title

CRISPR Correction of Duchenne Muscular Dystrophy (English)

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

1 May 2020

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Duchenne muscular dystrophy

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Rhonda Bassel-Duby

2

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Eric N. Olson

object named as

Eric N Olson

series ordinal

3

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author name string

Yi-Li Min

series ordinal

1

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

1 May 2020

language of work or name

English

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publication date

31 October 2018

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

Annual Review of Medicine

1 May 2020

published in

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

1 May 2020

volume

70

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

1 May 2020

page(s)

239-255

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy

1 May 2020

cites work

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12 December 2020

The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy

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12 December 2020

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

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12 December 2020

Recovery of induced mutations for X chromosome-linked muscular dystrophy in mice

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12 December 2020

Canine X-linked muscular dystrophy. An animal model of Duchenne muscular dystrophy: clinical studies

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12 December 2020

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

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12 December 2020

Muscular dystrophy in the mdx mouse: histopathology of the soleus and extensor digitorum longus muscles.

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12 December 2020

Progressive muscular dystrophy in a golden retriever dog: light microscope and ultrastructural features at 4 and 8 months

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12 December 2020

Muscle development in mdx mutant mice

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12 December 2020

X chromosome-linked muscular dystrophy (mdx) in the mouse

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12 December 2020

Primary progressive muscular dystrophy in the dog

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12 December 2020

Phenotype of dystrophinopathy in old MDX mice

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12 December 2020

mdx mice show progressive weakness and muscle deterioration with age

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12 December 2020

The structural and functional diversity of dystrophin

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12 December 2020

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

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12 December 2020

Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

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12 December 2020

Skeletal and Cardiac Myopathies in Mice Lacking Utrophin and Dystrophin: A Model for Duchenne Muscular Dystrophy

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12 December 2020

Dystrophin nonsense mutation induces different levels of exon 29 skipping and leads to variable phenotypes within one BMD family

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12 December 2020

A new model mouse for Duchenne muscular dystrophy produced by 2.4 Mb deletion of dystrophin gene using Cre-loxP recombination system

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12 December 2020

Absence of alpha 7 integrin in dystrophin-deficient mice causes a myopathy similar to Duchenne muscular dystrophy

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12 December 2020

Severe muscular dystrophy in mice that lack dystrophin and alpha7 integrin

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12 December 2020

Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule

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12 December 2020

Dystrophin-deficient mdx mice display a reduced life span and are susceptible to spontaneous rhabdomyosarcoma

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12 December 2020

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

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12 December 2020

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery

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12 December 2020

Cardiomyopathy in Duchenne muscular dystrophy: pathogenesis and therapeutics

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12 December 2020

A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping

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12 December 2020

An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breed

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12 December 2020

Facioscapulohumeral dystrophy: incomplete suppression of a retrotransposed gene

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12 December 2020

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

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12 December 2020

A functionally characterized test set of human induced pluripotent stem cells

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12 December 2020

Reference Maps of human ES and iPS cell variation enable high-throughput characterization of pluripotent cell lines

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12 December 2020

The passive mechanical properties of the extensor digitorum longus muscle are compromised in 2- to 20-mo-old mdx mice

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12 December 2020

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

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12 December 2020

Multiplex genome engineering using CRISPR/Cas systems

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12 December 2020

RNA-guided human genome engineering via Cas9

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12 December 2020

Role of telomere dysfunction in cardiac failure in Duchenne muscular dystrophy

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12 December 2020

RNA-guided gene activation by CRISPR-Cas9-based transcription factors

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12 December 2020

Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis

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12 December 2020

Differences in carrier frequency between mothers of Duchenne and Becker muscular dystrophy patients

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12 December 2020

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

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12 December 2020

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

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12 December 2020

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

Duchenne muscular dystrophy gene therapy in the canine model

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12 December 2020

Duchenne muscular dystrophy gene therapy in the canine model.

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12 December 2020

Highly efficient Cas9-mediated transcriptional programming

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12 December 2020

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

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12 December 2020

In vivo genome editing using Staphylococcus aureus Cas9

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12 December 2020

Exon skipping therapy for Duchenne muscular dystrophy

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12 December 2020

The Dystrophin Complex: Structure, Function, and Implications for Therapy

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12 December 2020

Engineering Human Stem Cell Lines with Inducible Gene Knockout using CRISPR/Cas9

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12 December 2020

The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development

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12 December 2020

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

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12 December 2020

CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy

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12 December 2020

Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

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

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12 December 2020

Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy

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12 December 2020

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

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12 December 2020

Satellite Cells in Muscular Dystrophy - Lost in Polarity.

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12 December 2020

A Human Pluripotent Stem Cell Model of Facioscapulohumeral Muscular Dystrophy-Affected Skeletal Muscles.

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12 December 2020

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model.

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12 December 2020

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

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12 December 2020

Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease

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12 December 2020

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

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12 December 2020

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

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12 December 2020

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA.

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12 December 2020

CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing.

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12 December 2020

Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies

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12 December 2020

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

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12 December 2020

Myotonic dystrophy type 1 patient-derived iPSCs for the investigation of CTG repeat instability.

1 reference

12 December 2020

Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration.

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12 December 2020

The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD.

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12 December 2020

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

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12 December 2020

Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy

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12 December 2020

Functional correction of dystrophin actin binding domain mutations by genome editing

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12 December 2020

Induced Pluripotent Stem Cells derived Muscle Progenitors Effectively Mitigate Muscular Dystrophy through Restoring the Dystrophin Distribution

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12 December 2020

Generation of an induced pluripotent stem cell line (CSCRMi001-A) from a patient with a new type of limb-girdle muscular dystrophy (LGMD) due to a missense mutation in POGLUT1 (Rumi).

1 reference

12 December 2020

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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12 December 2020

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

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12 December 2020

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

1 reference

12 December 2020

In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease

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12 December 2020

Duchenne Muscular Dystrophy (DMD): An updated review of common available therapies.

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12 December 2020

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

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12 December 2020

CRISPR/Cas9 cleavages in budding yeast reveal templated insertions and strand-specific insertion/deletion profiles

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12 December 2020

A dystrophic Duchenne mouse model for testing human antisense oligonucleotides.

1 reference

12 December 2020

Duchenne and Becker muscular dystrophy in adolescents: current perspectives.

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12 December 2020

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

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12 December 2020

Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing

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12 December 2020

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA induces homology-directed DNA repair

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12 December 2020

The 2018 version of the gene table of monogenic neuromuscular disorders (nuclear genome)

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12 December 2020

CRISPR-SKIP: programmable gene splicing with single base editors

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12 December 2020

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

1 reference

12 December 2020

10.1146/ANNUREV-MED-081117-010451

Identifiers

DOI

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

1 May 2020

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30379597 AND SRC:MED&resulttype=core&format=json

1 May 2020

PubMed publication ID

30379597

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