Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy (Q93376356)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

title

Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

Duchenne muscular dystrophy

5 May 2020

main subject

1 reference

0 references

0 references

1 reference

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

Toshifumi Yokota

series ordinal

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

author name string

Chantal Yoon

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

publication date

24 November 2018

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

Journal of personalized medicine

5 May 2020

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

volume

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

issue

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

Creative Commons Attribution 4.0 International

5 May 2020

copyright license

start time

24 November 2018

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

0 references

cites work

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CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo

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A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy

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Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping

11 February 2021

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

11 February 2021

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Cas9 immunity creates challenges for CRISPR gene editing therapies

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Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

11 February 2021

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CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response

11 February 2021

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p53 inhibits CRISPR-Cas9 engineering in human pluripotent stem cells

11 February 2021

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A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9.

11 February 2021

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

11 February 2021

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Delivering CRISPR: a review of the challenges and approaches

11 February 2021

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CRISPR off-target analysis in genetically engineered rats and mice

11 February 2021

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Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

11 February 2021

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

11 February 2021

1 reference

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Determination of qPCR Reference Genes Suitable for Normalizing Gene Expression in a Canine Model of Duchenne Muscular Dystrophy.

11 February 2021

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SaCas9 Requires 5'-NNGRRT-3' PAM for Sufficient Cleavage and Possesses Higher Cleavage Activity than SpCas9 or FnCpf1 in Human Cells.

11 February 2021

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

11 February 2021

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A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping

11 February 2021

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Skipping Multiple Exons to Treat DMD-Promises and Challenges.

11 February 2021

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Improving CRISPR-Cas specificity with chemical modifications in single-guide RNAs

11 February 2021

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CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes

11 February 2021

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

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Viral Vectors, Engineered Cells and the CRISPR Revolution

11 February 2021

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Immunity to CRISPR Cas9 and Cas12a therapeutics

11 February 2021

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Functional correction of dystrophin actin binding domain mutations by genome editing

11 February 2021

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

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

11 February 2021

1 reference

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

11 February 2021

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CRISPR-Cas9 Structures and Mechanisms

11 February 2021

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Eteplirsen in the treatment of Duchenne muscular dystrophy

11 February 2021

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Highly efficient RNA-guided base editing in mouse embryos

11 February 2021

1 reference

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

11 February 2021

1 reference

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Cas9, Cpf1 and C2c1/2/3-What's next?

11 February 2021

1 reference

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Porcine Zygote Injection with Cas9/sgRNA Results in DMD-Modified Pig with Muscle Dystrophy

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Current and emerging treatment strategies for Duchenne muscular dystrophy

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

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

11 February 2021

1 reference

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

11 February 2021

1 reference

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

11 February 2021

1 reference

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

11 February 2021

1 reference

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Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications.

11 February 2021

1 reference

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Cardiomyocytes from human pluripotent stem cells: From laboratory curiosity to industrial biomedical platform.

11 February 2021

1 reference

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Dystrophin induced cognitive impairment: mechanisms, models and therapeutic strategies

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9.

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

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

11 February 2021

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

11 February 2021

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Urine-derived stem cells: A novel and versatile progenitor source for cell-based therapy and regenerative medicine

11 February 2021

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Programmable RNA recognition and cleavage by CRISPR/Cas9.

11 February 2021

1 reference

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Generation of muscular dystrophy model rats with a CRISPR/Cas system

11 February 2021

1 reference

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Development and applications of CRISPR-Cas9 for genome engineering

11 February 2021

1 reference

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DNA interrogation by the CRISPR RNA-guided endonuclease Cas9.

11 February 2021

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Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells.

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

RNA-guided human genome engineering via Cas9

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Multiplex genome engineering using CRISPR/Cas systems

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Generation of human induced pluripotent stem cells from urine samples.

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

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Evidence-based path to newborn screening for Duchenne muscular dystrophy

11 February 2021

1 reference

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CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Best Practice Guidelines on molecular diagnostics in Duchenne/Becker muscular dystrophies

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Short motif sequences determine the targets of the prokaryotic CRISPR defence system

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Update on the management of Duchenne muscular dystrophy

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Optimizing exon skipping therapies for DMD.

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Characterizing interspecies uncertainty using data from studies of anti-neoplastic agents in animals and humans

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Generation and Characterization of Transgenic Mice with the Full-length HumanDMDGene

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Induction of pluripotent stem cells from adult human fibroblasts by defined factors

11 February 2021

1 reference

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Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy

11 February 2021

1 reference

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Mouse models of long QT syndrome

11 February 2021

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Dystrophin, its interactions with other proteins, and implications for muscular dystrophy

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Studying cardiac arrhythmias in the mouse--a reasonable model for probing mechanisms?

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Dystrophin and mutations: one gene, several proteins, multiple phenotypes

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer.

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Non-toxic ubiquitous over-expression of utrophin in the mdx mouse

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Dystrophin and muscular dystrophy: past, present, and future

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Exon structure of the human dystrophin gene

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Association of dystrophin and an integral membrane glycoprotein.

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

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

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Immunolocalization and developmental expression of dystrophin related protein in skeletal muscle.

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

Population frequencies of inherited neuromuscular diseases—A world survey

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

11 February 2021

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6313657

11 February 2021

Identifiers

DOI

10.3390/JPM8040038

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30477208 AND SRC:MED&resulttype=core&format=json

5 May 2020

PubMed publication ID

30477208

1 reference