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

Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease

16 May 2024

Abstract excerpt

Gaucher Disease (GD) is an inherited metabolic disorder caused by mutations in the GBA1 gene. It can manifest with severe neurodegeneration and visceral pathology. The most acute neuronopathic form (nGD), for which there are no curative therapeutic options, is characterised by devastating neuropathology and death during infancy. In this study, we investigated the therapeutic benefit of systemically delivered AAV9...

Read the complete abstract on PubMed

Topics

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

Share to Autism Spectrum Disorder Research
Share to Cellular transport and secretion
Share to Lysosomal Storage Disorders Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 38757200
DOI: 10.1093/hmg/ddae081

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher diseasePMID 38757200Read ↗

Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytesSemantic proximity Read ↗

Development of a Lentiviral Vector for High-Yield Production of Synthetic and Recombinant GCase for Gaucher Disease TherapySemantic proximity Read ↗

AAV delivery of <i>GBA1</i> suppresses α-synuclein accumulation in Parkinson’s disease models and restores motor dysfunction in a Gaucher’s disease modelSemantic proximity Read ↗

AAV delivery of GBA1 suppresses α-synuclein accumulation in Parkinson’s disease models and restores functions in Gaucher’s disease modelsSemantic proximity Read ↗

AAV gene therapy for GBA-PD and Gaucher DiseaseSemantic proximity Read ↗

Delivery of Glucosylceramidase Beta Gene Using AAV9 Vector Therapy as a Treatment Strategy in Mouse Models of Gaucher DiseaseSemantic proximity Read ↗

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

Advancements in Viral Gene Therapy for Gaucher DiseaseSemantic proximity Read ↗

Intrinsic link between PGRN and Gba1 D409V mutation dosage in potentiating Gaucher disease.Semantic proximity Read ↗

Glucocerebrosidase as a therapeutic target for Parkinson's disease.Semantic proximity Read ↗

AAV gene therapy for GBA1-related diseases.Semantic proximity Read ↗

Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse modelSemantic proximity Read ↗

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