[HTML][HTML] Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin
ML Sosulski, KM Stiles, EZ Frenk, FM Hart… - JCI insight, 2020 - ncbi.nlm.nih.gov
Abstract Alpha 1-antitrypsin (AAT) deficiency, a hereditary disorder characterized by low
serum levels of functional AAT, is associated with early development of panacinar …
serum levels of functional AAT, is associated with early development of panacinar …
Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency
JB Rosenberg, BP De, A Greco, N Gorman… - Human Gene …, 2023 - liebertpub.com
α1-antitrypsin (AAT) deficiency is a common autosomal recessive hereditary disorder, with a
high risk for the development of early-onset panacinar emphysema. AAT, produced primarily …
high risk for the development of early-onset panacinar emphysema. AAT, produced primarily …
Alpha1-antitrypsin deficiency
RT Abboud - Respiratory Medicine: COPD Update, 2006 - Elsevier
Severe alpha1-antitrypsin (AAT) deficiency is an inherited disorder, leading to the
development of emphysema in smokers at a relatively young age and disability in most …
development of emphysema in smokers at a relatively young age and disability in most …
Historical role of alpha-1-antitrypsin deficiency in respiratory and hepatic complications
Abstract Alpha-1-antitrypsin (AAT) deficiency is a heritable disease that is commonly
associated with complications in the respiratory and hepatic systems. AAT acts as a …
associated with complications in the respiratory and hepatic systems. AAT acts as a …
Gene therapy for alpha-1 antitrypsin deficiency lung disease
MJ Chiuchiolo, RG Crystal - Annals of the American Thoracic …, 2016 - atsjournals.org
Alpha-1 antitrypsin (AAT) deficiency, characterized by low plasma levels of the serine
protease inhibitor AAT, is associated with emphysema secondary to insufficient protection of …
protease inhibitor AAT, is associated with emphysema secondary to insufficient protection of …
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques
M Blackwood, AM Gruntman, Q Tang… - … Therapy Methods & …, 2024 - cell.com
Alpha-1 antitrypsin deficiency (AATD) is characterized by both chronic lung disease due to
loss of wild-type AAT (M-AAT) antiprotease function and liver disease due to toxicity from …
loss of wild-type AAT (M-AAT) antiprotease function and liver disease due to toxicity from …
[HTML][HTML] Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease
CI Wooddell, K Blomenkamp, RM Peterson… - JCI insight, 2020 - ncbi.nlm.nih.gov
The autosomal codominant genetic disorder alpha-1 antitrypsin (AAT) deficiency (AATD)
causes pulmonary and liver disease. Individuals homozygous for the mutant Z allele …
causes pulmonary and liver disease. Individuals homozygous for the mutant Z allele …
Alpha-1-antitrypsin augmentation therapy for alpha-1-antitrypsin deficiency
RC Hubbard, RG Crystal - The American journal of medicine, 1988 - Elsevier
Alpha-1-antitrypsin (A1AT) deficiency is a genetic disorder characterized by low serum
levels of A1AT and a high risk for the development of emphysema. A1AT is the principal …
levels of A1AT and a high risk for the development of emphysema. A1AT is the principal …
[HTML][HTML] Ferret models of alpha-1 antitrypsin deficiency develop lung and liver disease
N He, X Liu, AR Vegter, TIA Evans, JS Gray, J Guo… - JCI insight, 2022 - ncbi.nlm.nih.gov
Abstract Alpha-1 antitrypsin deficiency (AATD) is the most common genetic cause and risk
factor for chronic obstructive pulmonary disease, but the field lacks a large-animal model …
factor for chronic obstructive pulmonary disease, but the field lacks a large-animal model …
Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles
C Mueller, Q Tang, A Gruntman, K Blomenkamp… - Molecular Therapy, 2012 - cell.com
α-1 antitrypsin (AAT) deficiency can exhibit two pathologic states: a lung disease that is
primarily due to the loss of AAT's antiprotease function, and a liver disease resulting from a …
primarily due to the loss of AAT's antiprotease function, and a liver disease resulting from a …