Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin–related liver disease in mice

Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B

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Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

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Liver Test Results Do Not Identify Liver Disease in Adults With α1-Antitrypsin Deficiency

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Preventing and reversing the cellular consequences of Z alpha-1 antitrypsin accumulation by targeting s4A

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Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

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(2011) Robert Bals   BEST PRACTICE & RESEARCH IN CLINICAL GASTROENTEROLOGY

Diagnosis and Management of Patients With α1-Antitrypsin (A1AT) Deficiency

(2011) David R. Nelson et al.   Clinical Gastroenterology and Hepatology

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

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Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

(2011) Kosuke Yusa et al.   NATURE

Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

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The discovery of α1-antitrypsin and its role in health and disease

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Hepatic Fibrosis and Carcinogenesis in  1-Antitrypsin Deficiency: A Prototype for Chronic Tissue Damage in Gain-of-Function Disorders

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RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Characteristics of hepatocellular carcinoma in a murine model of alpha-1-antitrypsin deficiency

(2010) Nancy Y. Marcus et al.   HEPATOLOGY RESEARCH

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

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Alpha1-Antitrypsin Deficiency and Autophagy

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Defining the mechanism of polymerization in the serpinopathies

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An Autophagy-Enhancing Drug Promotes Degradation of Mutant  1-Antitrypsin Z and Reduces Hepatic Fibrosis

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α1-Antitrypsin deficiency, chronic obstructive pulmonary disease and the serpinopathies

(2009) Ugo I. Ekeowa et al.   CLINICAL SCIENCE

Mechanisms of emphysema in  1-antitrypsin deficiency: molecular and cellular insights

(2009) B. Gooptu et al.   EUROPEAN RESPIRATORY JOURNAL

Alpha1-Antitrypsin Deficiency

(2009) Edwin K. Silverman et al.   NEW ENGLAND JOURNAL OF MEDICINE

Liver Disease in Alpha 1-Antitrypsin Deficiency: A Review

(2008) Kyrsten D. Fairbanks et al.   AMERICAN JOURNAL OF GASTROENTEROLOGY

Screening and Familial Testing of Patients for α 1 -Antitrypsin Deficiency

(2008) D. Kyle Hogarth et al.   CHEST

Alpha-1 antitrypsin deficiency: A conformational disease associated with lung and liver manifestations

(2008) C. M. Greene et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Hereditary alpha-1-antitrypsin deficiency and its clinical consequences

(2008) Laura Fregonese et al.   Orphanet Journal of Rare Diseases

Alpha-1 antitrypsin Null mutations and severity of emphysema

(2008) Laura Fregonese et al.   RESPIRATORY MEDICINE