Sage Journals: Discover world-class research

Free access

Review article

First published 2012-10

Prevalence of α1-antitrypsin deficiency alleles PIS and PIZ worldwide and effective screening for each of the five phenotypic classes PIMS,PIMZ,PISS,PISZ,and PI*ZZ: a comprehensive review

Frederick J. de Serres, Ignacio Blanco

Therapeutic Advances in Respiratory Disease

Free access

Research article

First published 2012-4

Rare alpha-1-antitrypsin variants: are they really so rare?

Francisco Rodriguez-Frias, Marc Miravitlles, Rafael Vidal [...]

Therapeutic Advances in Respiratory Disease

Free access

Review article

First published 2014-10

α1 Antitrypsin deficiency: current best practice in testing and augmentation therapy

Michael A. Campos, Jorge Lascano

Therapeutic Advances in Respiratory Disease

Free access

Research article

First published 2018-6

Liver disease in adults with α1-antitrypsin deficiency

Mattias Mandorfer, Theresa Bucsics, Veronika Hutya [...]

United European Gastroenterology Journal

Retricted access

Research article

First published 2023-02

Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency

Jonathan B. Rosenberg, Bishnu P. De, Alessandria Greco [...]

Human Gene Therapy

Retricted access

Research article

First published 2013-12

Intrapleural Administration of an AAVrh.10 Vector Coding for Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency

Maria J. Chiuchiolo, Stephen M. Kaminsky, Dolan Sondhi [...]

Human Gene Therapy Clinical Development

Retricted access

Research article

First published 2014-09

Phase I/II Study of Intrapleural Administration of a Serotype rh.10 Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human α1-Antitrypsin cDNA to Individuals with α1-Antitrypsin Deficiency

Maria J. Chiuchiolo, Stephen M. Kaminsky, Dolan Sondhi [...]

Human Gene Therapy Clinical Development

Retricted access

Research article

First published 2008-6

Correction: Intravenous Human Plasma–Derived Augmentation Therapy in α1-Antitrypsin Deficiency: From Pharmacokinetic Analysis to Individualizing Therapy

Annals of Pharmacotherapy

Retricted access

Review article

First published 2017-01

Genetic Modification of the Lung Directed Toward Treatment of Human Disease

Dolan Sondhi, Katie M. Stiles, Bishnu P. De [...]

Human Gene Therapy

Open access

Research article

First published 2007

Chemical Chaperone and Inhibitor Discovery: Potential Treatments for Protein Conformational Diseases

Jian-Hua Zhao, Hsuan-Liang Liu, Hsin-Yi Lin [...]

Perspectives in Medicinal Chemistry

Search results