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

Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides.

EBioMedicine - 1 Dec 2020

Vila-Julià Ferran, Cabrera-Pérez Raquel, Cámara Yolanda, Molina-Berenguer Miguel, Lope-Piedrafita Silvia, Hirano Michio, Mingozzi Federico, Torres-Torronteras Javier, Martí Ramon

Abstract excerpt

BACKGROUND: Preclinical studies have shown that gene therapy is a feasible approach to treat mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). However, the genetic murine model of the disease (Tymp/Upp1 double knockout, dKO) has a limited functional phenotype beyond the metabolic imbalances, and so the studies showing efficacy of gene therapy have relied almost exclusively on demonstrating correction...

Read the complete abstract on PubMed

Topics

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

Share to Combined Modality Therapy
Share to Dependovirus

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Intestinal Pseudo-Obstruction

Share to Mitochondrial Diseases

Share to Muscular Dystrophy, Oculopharyngeal

Share to Nucleosides

Share to Ophthalmoplegia

Share to Thymidine Phosphorylase

Source

Open in PubMed Open full text

Identifiers

PMID: 33232869
DOI: 10.1016/j.ebiom.2020.103133
PMCID: PMC7689515

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides.PMID 33232869Read ↗

Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE.Semantic proximity Read ↗

Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal EncephalomyopathySemantic proximity Read ↗

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): biochemical features and therapeutic approaches.Semantic proximity Read ↗

Thymidine phosphorylase deficiency causes MNGIE: an autosomal recessive mitochondrial disorder.Semantic proximity Read ↗

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): Position paper on diagnosis, prognosis, and treatment by the MNGIE International Network.Semantic proximity Read ↗

Targeted deletion of both thymidine phosphorylase and uridine phosphorylase and consequent disorders in mice.Semantic proximity Read ↗

GENE THERAPY PREVENTS HEPATIC MITOCHONDRIAL DYSFUNCTION IN MURINE DEOXYGUANOSINE KINASE DEFICIENCYSemantic proximity Read ↗

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

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of MetabolismSemantic proximity Read ↗

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

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

Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.Semantic proximity Read ↗

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