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

Lentivirus-mediated gene therapy for Fabry disease

25 Feb 2021

Abstract excerpt

Abstract Enzyme and chaperone therapies are used to treat Fabry disease. Such treatments are expensive and require intrusive biweekly infusions; they are also not particularly efficacious. In this pilot, single-arm study (NCT02800070), five adult males with Type 1 (classical) phenotype Fabry disease were infused with autologous lentivirus-transduced, CD34 + -selected, hematopoietic stem/progenitor cells...

Read the complete abstract on PubMed

Topics

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

Share to Cellular transport and secretion
Share to Cytomegalovirus and herpesvirus research
Share to Lysosomal Storage Disorders Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 33633114
DOI: 10.1038/s41467-021-21371-5

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

Lentivirus-mediated gene therapy for Fabry diseasePMID 33633114Read ↗

Therapeutic advances in Fabry disease: The future awaits.Semantic proximity Read ↗

Current and Investigational Therapeutics for Fabry DiseaseSemantic proximity Read ↗

Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing.Semantic proximity Read ↗

Chaperone Therapy in Fabry Disease.Semantic proximity Read ↗

Anderson–Fabry Disease: From Endothelial Dysfunction to Emerging TherapiesSemantic proximity Read ↗

Fabry disease revisited: Management and treatment recommendations for adult patientsSemantic proximity Read ↗

Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA approval.Semantic proximity Read ↗

Developments in the treatment of Fabry disease.Semantic proximity Read ↗

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studiesSemantic proximity Read ↗

Precision medicine in Fabry disease.Semantic proximity Read ↗

Genetics and Gene Therapy of Anderson-Fabry Disease.Semantic proximity Read ↗

Fabry disease: Mechanism and therapeutics strategiesSemantic proximity Read ↗

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