نتایج جستجو برای: 1 antitrypsin deficiency

تعداد نتایج: 2853876  

Journal: :Molecular therapy : the journal of the American Society of Gene Therapy 2017
Christian Mueller Gwladys Gernoux Alisha M Gruntman Florie Borel Emer P Reeves Roberto Calcedo Farshid N Rouhani Anthony Yachnis Margaret Humphries Martha Campbell-Thompson Louis Messina Jeffrey D Chulay Bruce Trapnell James M Wilson Noel G McElvaney Terence R Flotte

Alpha-1 antitrypsin deficiency is a monogenic disorder resulting in emphysema due principally to the unopposed effects of neutrophil elastase. We previously reported achieving plasma wild-type alpha-1 antitrypsin concentrations at 2.5%-3.8% of the purported therapeutic level at 1 year after a single intramuscular administration of recombinant adeno-associated virus serotype 1 alpha-1 antitrypsi...

2012
Mun Peak Nyon John Kirkpatrick Lisa D. Cabrita John Christodoulou Bibek Gooptu

Alpha(1)-antitrypsin is a 45-kDa (394-residue) serine protease inhibitor synthesized by hepatocytes, which is released into the circulatory system and protects the lung from the actions of neutrophil elastase via a conformational transition within a dynamic inhibitory mechanism. Relatively common point mutations subvert this transition, causing polymerisation of α(1)-antitrypsin and deficiency ...

2017
Newton Key Hokama Marcelo Padovani de Toledo Moraes Paula de Oliveira Montandon Hokama Fernando Gomes Romeiro

Most patients with alpha1 antitrypsin deficiency do not receive this diagnosis until developing severe complications, in particular when respiratory symptoms are absent. This is a reason for making alpha1 antitrypsin deficiency a possible diagnosis among patients with cryptogenic cirrhosis or other conditions of liver disease without a clear etiology. In this report, a case of cryptogenic cirrh...

2015
OF McElvaney L Fee C O’Connor TP Carroll

Background Alpha-1 antitrypsin deficiency (AATD) is a hereditary disorder defined by low plasma levels of alpha-1 antitrypsin (AAT). It is linked primarily with the development of lung, liver and skin disease. The most common abnormal variant of AAT is the ‘Z’ variant. It is the AATD type most associated with the development of liver disease. The aim of this project is to determine the prevalen...

2017
Meredith Corley Amanda Solem Gabriela Phillips Lela Lackey Benjamin Ziehr Heather A Vincent Anthony M Mustoe Silvia B V Ramos Kevin M Weeks Nathaniel J Moorman Alain Laederach

Chronic obstructive pulmonary disease (COPD) affects over 65 million individuals worldwide, where α-1-antitrypsin deficiency is a major genetic cause of the disease. The α-1-antitrypsin gene, SERPINA1, expresses an exceptional number of mRNA isoforms generated entirely by alternative splicing in the 5'-untranslated region (5'-UTR). Although all SERPINA1 mRNAs encode exactly the same protein, ex...

Journal: :American journal of respiratory and critical care medicine 2003
J K Stoller G L Snider M L Brantly R J Fallat R A Stockley G M Turino N Konietzko A Dirksen E Eden M Luisetti J Stolk C Strange

Introduction . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 820 Goals, Organization of the Project, and Timeline . . . . . 820 Summary of Main Recommendations Regarding Diagnosis and Management by the Alpha-1 Antitrypsin Deficiency Task Force . . . . . . . . . . . . . 820 Clinical Recognition of AAT Deficiency . . . . . . . . . . 820 Genetic Testing for AAT Deficiency . ....

1998
Ravi Mahadeva David A Lomas

Emphysema is a chronic progressive lung disease characterised by abnormal permanent enlargement of airspaces as a result of destruction of alveolar walls. Most patients develop emphysema as a consequence of smoking but 1–2% of patients with emphysema develop the condition as a result of a genetic deficiency of the plasma proteinase inhibitor á1-antitrypsin. The two common deficiency variants of...

2017
Roberto Calcedo Suryanarayan Somanathan Qiuyue Qin Michael R. Betts Andrew J. Rech Robert H. Vonderheide Christian Mueller Terence R. Flotte James M. Wilson

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