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

Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency.

The New England journal of medicine - 11 Aug 2022

Strnad Pavel, Mandorfer Mattias, Choudhury Gourab, Griffiths William, Trautwein Christian, Loomba Rohit, Schluep Thomas, Chang Ting, Yi Min, Given Bruce D, Hamilton James C, San Martin Javier, Teckman Jeffery H

Abstract excerpt

BACKGROUND: Alpha1-antitrypsin (AAT) deficiency results from carriage of a homozygous SERPINA1 "Z" mutation (proteinase inhibitor [PI] ZZ). The Z allele produces a mutant AAT protein called Z-AAT, which accumulates in hepatocytes and can lead to progressive liver disease and fibrosis. This open-label, phase 2 trial investigated the safety and efficacy of fazirsiran, an RNA interference therapeutic, in patients...

Read the complete abstract on PubMed

Topics

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

Share to alpha 1-Antitrypsin
Share to alpha 1-Antitrypsin Deficiency

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Genotype

Share to Hepatocytes

Share to Injections, Subcutaneous

Share to Liver Cirrhosis

Share to Mutation

Source

Identifiers

PMID: 35748699
DOI: 10.1056/NEJMoa2205416

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

Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency.PMID 35748699Read ↗

Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.Semantic proximity Read ↗

Alpha-1 antitrypsin deficiency-associated liver disease: From understudied disorder to the poster child of genetic medicineSemantic proximity Read ↗

Cleaning up alpha-1 antitrypsin deficiency related liver disease.Semantic proximity Read ↗

Pediatric and Adult Liver Disease in Alpha-1 Antitrypsin Deficiency.Semantic proximity Read ↗

Alpha1-antitrypsin deficiency: New therapies on the horizon.Semantic proximity Read ↗

Liver biopsy confirms precise and efficient correction of SERPINA1 after <i>in vivo</i> Base Editing in a Patient with Alpha-1 Antitrypsin DeficiencySemantic proximity Read ↗

α1-Antitrypsin deficiency.Semantic proximity Read ↗

Small molecule screen employing patient-derived iPSC hepatocytes identifies LRRK2 as a novel therapeutic target for Alpha1 Antitrypsin DeficiencySemantic proximity Read ↗

Liver disease in alpha-1 antitrypsin deficiency: current understanding and future therapy.Semantic proximity Read ↗

[Alpha-1-antitrypsin deficiency].Semantic proximity Read ↗

Alpha-1 antitrypsin and liver disease: mechanisms of injury and novel interventions.Semantic proximity Read ↗

Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.Semantic proximity Read ↗

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