Gene therapy for alpha-1 antitrypsin deficiency

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Gene therapy for alpha 1-antitrypsin deficiency.

Alpha 1-antitrypsin deficiency is associated with pulmonary emphysema and liver cirrhosis. The pathogenesis and treatment is reviewed with particular emphasis on gene therapy for emphysema. The technology of gene transfer using viruses and liposomes is developing fast and animal experiments have confirmed the feasibility of gene therapy for alpha 1-antitrypsin deficiency (AATD). So far only sub...

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Challenges and Prospects for Alpha-1 Antitrypsin Deficiency Gene Therapy.

Alpha-1 antitrypsin (AAT) is a protease inhibitor belonging to the serpin family. A number of identified mutations in the SERPINA1 gene encoding this protein result in alpha-1 antitrypsin deficiency (AATD). A decrease in AAT serum concentration or reduced biological activity causes considerable risk of chronic respiratory and liver disorders. As a monogenic disease, AATD appears to be an attrac...

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Augmentation therapy in alpha(1)-antitrypsin deficiency.

alpha(1)-Antitrypsin (AAT) deficiency is a common but under-recognised condition. Since its first description by Laurell and Eriksson in 1963, significant advances have been made in understanding the genetics, physiology and pathophysiology of this condition. The intravenous administration of purified AAT to AAT-deficient individuals has been shown to confer biochemical efficacy by raising the ...

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Alpha 1 antitrypsin deficiency.

Alpha-1 antitrypsin deficiency is an inherited disorder that may cause severe lung and liver disease.

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Therapy for Alpha-1 Antitrypsin Deficiency: the Evidence for Efficacy

Disclosure: David Parr has received honoraria for advising CSL Behring and Grifols (formerly Talecris), in addition to sponsorship from AstraZeneca and Boehringer Ingelheim. Emer Reeves and Niels Seersholm have no competing interests to disclose; however, both have received honoraria for this educational activity funded by CSL Behring. Kenneth R. Chapman has received compensation for consulting...

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ژورنال

عنوان ژورنال: Human Molecular Genetics

سال: 2011

ISSN: 0964-6906,1460-2083

DOI: 10.1093/hmg/ddr156