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Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

Molecular Therapy(2021)10.1016/j.ymthe.2021.09.003Source: DataRank Database

Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy is a research paper published in Molecular Therapy (2021). On theSindex it has a DataRank of 0. It has been cited 67 times.

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Open Access67 citations

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Enrichment:Funding (10 grants)OA: hybridTopics (26)IDs (PubMed, PMC)Annotations (1)

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Findable (1/2)

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Accessible (1/2)

Open Access

Interoperable (0/2)

Reusable (0/3)

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Authors (10)

Veronica Gough ORCID,Joel D. Bohning,Siyan Liu ORCID,Jacqueline N. Robinson-Hamm ORCID,Heather Daniels

Live enrichment

PubMed:34509668 PMC:PMC8571168

NIBIB NIH HHS

Grant: U01 EB028901

NIGMS NIH HHS

Grant: T32 GM008555

NCATS NIH HHS

Grant: UL1 TR002553

NIAMS NIH HHS

Grant: UG3 AR075336

NIGMS NIH HHS

Grant: T32 GM144291

NIAMS NIH HHS

Grant: UH3 AR075336

NIAMS NIH HHS

Grant: R01 AR069085

NHLBI NIH HHS

Grant: U01 HL156348

National Institutes of Health

Office of the Assistant Secretary of Defense for Health Affairs

hybridLicense: cc-by-nc-nd

publisherhttp://www.cell.com/article/S1525001621004585/pdf

publisherhttps://api.elsevier.com/content/article/PII:S1525001621004585?httpAccept=text/xml

publisherhttps://api.elsevier.com/content/article/PII:S1525001621004585?httpAccept=text/plain

repositoryhttps://www.ncbi.nlm.nih.gov/pmc/articles/8571168

Europe_PMChttps://europepmc.org/articles/PMC8571168

MeSH Terms

Muscle, SkeletalMyocardiumAnimalsMice, TransgenicHumansMiceDependovirusMuscular Dystrophy, DuchenneDisease Models, AnimalDystrophinGene Transfer TechniquesGenetic EngineeringVirus IntegrationGene ExpressionGene OrderMutationGenetic VectorsExonsGenetic TherapyCRISPR-Cas SystemsGene Editing

Keywords

Muscular dystrophytargeted integrationViral VectorsCrisprGenome Editing