Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1

24 Jun 2021

Abstract excerpt

Limb girdle muscular dystrophy (LGMD) 2A/R1, caused by mutations in the CAPN3 gene and CAPN3 loss of function, is known to play a role in disease pathogenicity. In this study, AAVrh74.tMCK.CAPN3 was delivered systemically to two different age groups of CAPN3 knockout (KO) mice; each group included two treatment cohorts receiving low (1.17 × 10 14 vg/kg) and high (2.35 × 10 14 vg/kg) doses of the vector and...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Calpain Protease Function and Regulation
Share to Muscle Physiology and Disorders
Share to Viral Infections and Immunology Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 34514031
DOI: 10.1016/j.omtm.2021.06.010

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1PMID 34514031Read ↗

AAV-TNNI3 rescues an experimental murine Tnni3 mutation resulting in thin filament mediated DCM.Semantic proximity Read ↗

Evidence for ACTN3 as a genetic modifier of Duchenne muscular dystrophy.Semantic proximity Read ↗

AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy modelsSemantic proximity Read ↗

Restriction of Calpain3 Expression to the Skeletal Muscle Prevents Cardiac Toxicity and Corrects Pathology in a Murine Model of Limb-Girdle Muscular DystrophySemantic proximity Read ↗

A Small-Molecule Approach to Restore a Slow-Oxidative Phenotype and Defective CaMKIIβ Signaling in Limb Girdle Muscular Dystrophy.Semantic proximity Read ↗

Limb-girdle muscular dystrophy 2A.Semantic proximity Read ↗

Alpha Protein Kinase 3 Gene Therapy Restores Heart Function in Mouse and Human Models of CardiomyopathySemantic proximity Read ↗

Missense mutation of c.635 T > C in CAPN3 impairs muscle injury repair in a Limb-Girdel Muscular Dystropy Model.Semantic proximity Read ↗

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse modelSemantic proximity Read ↗

Alpha protein kinase 3 gene therapy restores heart function in mouse and human models of cardiomyopathySemantic proximity Read ↗

Creatine and L-carnitine attenuate muscular laminopathy in the LMNA mutation transgenic zebrafish.Semantic proximity Read ↗

Different outcomes of endurance and resistance exercise in skeletal muscles of Oculopharyngeal muscular dystrophySemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.