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

AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease

2 Nov 2017

Abstract excerpt

Pompe disease (PD) is a metabolic myopathy due to acid alpha-glucosidase deficiency and characterized by extensive glycogen storage and impaired autophagy. We previously showed that modulation of autophagy and lysosomal exocytosis by overexpression of the transcription factor EB (TFEB) gene was effective in improving muscle pathology in PD mice injected intramuscularly with an AAV-TFEB vector. Here we have...

Read the complete abstract on PubMed

Topics

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

Share to Biochemical and Molecular Research
Share to Lysosomal Storage Disorders Research
Share to Trypanosoma species research and implications

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 29118420
DOI: 10.1038/s41598-017-15352-2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe DiseasePMID 29118420Read ↗

TFEB overexpression promotes glycogen clearance of Pompe disease iPSC-derived skeletal muscleSemantic proximity Read ↗

What’s new and what’s next for gene therapy in Pompe disease?Semantic proximity Read ↗

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.Semantic proximity Read ↗

Gene Therapy for Pompe Disease: The Time is nowSemantic proximity Read ↗

Pompe disease gene therapySemantic proximity Read ↗

Advancements in AAV-mediated Gene Therapy for Pompe DiseaseSemantic proximity Read ↗

Longitudinal characterization of Gaac.1826dupA mice reveals the cardiac, myopathic and biochemical phenotypes of Pompe disease.Semantic proximity Read ↗

Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapySemantic proximity Read ↗

A Comprehensive Update on Pompe Disease: From Existing Therapies to Emerging Curative Strategies.Semantic proximity Read ↗

A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe diseaseSemantic proximity Read ↗

Lysosomal glycogen accumulation in Pompe disease results in disturbed cytoplasmic glycogen metabolismSemantic proximity Read ↗

Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease miceSemantic proximity Read ↗

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