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Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human alpha1-Antitrypsin for the Treatment of alpha1-Antitrypsin Deficiency.
Rosenberg JB, De BP, Greco A, Gorman N, Kooner V, Chen A, Yost-Bido M, Munoz-Zuluaga C, Kaminsky SM, Rostami M, Monette S, Crystal RG, Sondhi D. Rosenberg JB, et al. Hum Gene Ther. 2023 Feb;34(3-4):139-149. doi: 10.1089/hum.2022.192. Hum Gene Ther. 2023. PMID: 36606685 Free PMC article.
alpha1-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 in the liver, functions to protect the lung from neutrophil protease; with AAT d …
alpha1-antitrypsin (AAT) deficiency is a common autosomal recessive hereditary disorder, with a high risk for the development of early-onset …
Intrapleural administration of an AAVrh.10 vector coding for human alpha1-antitrypsin for the treatment of alpha1-antitrypsin deficiency.
Chiuchiolo MJ, Kaminsky SM, Sondhi D, Hackett NR, Rosenberg JB, Frenk EZ, Hwang Y, Van de Graaf BG, Hutt JA, Wang G, Benson J, Crystal RG. Chiuchiolo MJ, et al. Hum Gene Ther Clin Dev. 2013 Dec;24(4):161-73. doi: 10.1089/humc.2013.168. Hum Gene Ther Clin Dev. 2013. PMID: 24191907
Serum alpha1AT levels <11 muM are associated with progressive destruction of lung parenchyma and early-onset of panacinar emphysema in the age range 35-45. The current approved treatment for alpha1AT deficiency is a costly protein augmentation therapy requ …
Serum alpha1AT levels <11 muM are associated with progressive destruction of lung parenchyma and early-onset of panacinar emphy …