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

Gene therapy for urea cycle defects: An update from historical perspectives to future prospects

7 Apr 2023

Abstract excerpt

Urea cycle defects (UCDs) are severe inherited metabolic diseases with high unmet needs which present a permanent risk of hyperammonaemic decompensation and subsequent acute death or neurological sequelae, when treated with conventional dietetic and medical therapies. Liver transplantation is currently the only curative option, but has the potential to be supplanted by highly effective gene therapy interventions...

Read the complete abstract on PubMed

Topics

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

Share to CRISPR and Genetic Engineering
Share to Viral gastroenteritis research and epidemiology
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 37026568
DOI: 10.1002/jimd.12609

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Gene therapy for urea cycle defects: An update from historical perspectives to future prospectsPMID 37026568Read ↗

The Evolution of Gene Therapy in the Treatment of Metabolic Liver Diseases.Semantic proximity Read ↗

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

Liver‐directed gene therapy for inherited metabolic diseasesSemantic proximity Read ↗

Gene Editing Correction of a Urea Cycle Defect in Organoid Stem Cell Derived Hepatocyte-like Cells.Semantic proximity Read ↗

Novel vectors and approaches for gene therapy in liver diseasesSemantic proximity Read ↗

Gene therapy in liver diseases: state-of-the-art and future perspectives.Semantic proximity Read ↗

Gene therapy for liver diseases — progress and challengesSemantic proximity Read ↗

Genetic-Based Approaches to Inherited Metabolic Liver DiseasesSemantic proximity Read ↗

Gene therapy for glycogen storage diseasesSemantic proximity Read ↗

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical DevelopmentSemantic proximity Read ↗

Gene therapy research for kidney diseasesSemantic proximity Read ↗

Liver directed adeno‐associated viral vectors to treat metabolic diseaseSemantic proximity Read ↗

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