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 AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

22 Feb 2017

Abstract excerpt

X-linked myotubular myopathy (XLMTM) results from MTM1 gene mutations and myotubularin deficiency. Most XLMTM patients develop severe muscle weakness leading to respiratory failure and death, typically within 2 years of age. Our objective was to evaluate the efficacy and safety of systemic gene therapy in the p.N155K canine model of XLMTM by performing a dose escalation study. A recombinant adeno-associated virus...

Read the complete abstract on PubMed

Topics

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

Share to Cardiomyopathy and Myosin Studies
Share to Muscle Physiology and Disorders
Share to Neurogenetic and Muscular Disorders Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 28237839
DOI: 10.1016/j.ymthe.2017.02.004

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 AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in DogsPMID 28237839Read ↗

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.Semantic proximity Read ↗

Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.Semantic proximity Read ↗

AAV-mediated gene transfer restores a normal muscle transcriptome in a canine model of X-linked myotubular myopathySemantic proximity Read ↗

A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in DogsSemantic proximity Read ↗

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophySemantic proximity Read ↗

Long-Term Systemic Myostatin Inhibition via Liver-Targeted Gene Transfer in Golden Retriever Muscular DystrophySemantic proximity Read ↗

Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular DystrophySemantic proximity Read ↗

Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice.Semantic proximity Read ↗

Refining gene delivery to skeletal muscle with a dual-strategy approach of muscle-tropic AAV capsids and muscle-specific promotersSemantic proximity Read ↗

Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne PatientsSemantic proximity Read ↗

Novel Muscle-Tropic AAV Capsids with Dramatically Enhanced Transduction and Safety Profiles in Non-Human PrimatesSemantic proximity Read ↗

Comparative in vivo characterization of newly discovered myotropic adeno-associated vectorsSemantic proximity Read ↗

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