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

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

Journal of hepatology - 1 Aug 2018

Turner Alice M, Stolk Jan, Bals Robert, Lickliter Jason D, Hamilton James, Christianson Dawn R, Given Bruce D, Burdon Jonathan G, Loomba Rohit, Stoller James K, Teckman Jeffery H

Abstract excerpt

BACKGROUND & AIMS: Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder causing pulmonary and liver disease. The PiZ mutation in AAT (SERPINA1) results in mis-folded AAT protein (Z-AAT) accumulating in hepatocytes, leading to fibrosis and cirrhosis. RNAi-based therapeutics silencing production of hepatic Z-AAT might benefit patients with AATD-associated liver disease. This study evaluated an RNAi...

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 Double-Blind Method

Share to Drug Carriers

Share to Drug Monitoring

Share to Healthy Volunteers

Share to Liver Cirrhosis

Share to Trypsin Inhibitors

Source

Identifiers

PMID: 29572094
DOI: 10.1016/j.jhep.2018.03.012

Genes

ARC
SERPINA1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.PMID 29572094Read ↗

Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency.Semantic proximity Read ↗

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.Semantic proximity Read ↗

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

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

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

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

Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice.Semantic proximity Read ↗

Knockdown of Z Mutant Alpha-1 Antitrypsin In Vivo Using Modified DNA Antisense Oligonucleotides.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 ↗

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

Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteinsSemantic proximity Read ↗

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