5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

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...

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Detalles Bibliográficos
Autores principales: Mueller, Christian, Gernoux, Gwladys, Gruntman, Alisha M., Borel, Florie, Reeves, Emer P., Calcedo, Roberto, Rouhani, Farshid N., Yachnis, Anthony, Humphries, Margaret, Campbell-Thompson, Martha, Messina, Louis, Chulay, Jeffrey D., Trapnell, Bruce, Wilson, James M., McElvaney, Noel G., Flotte, Terence R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5474959/
https://www.ncbi.nlm.nih.gov/pubmed/28408179
http://dx.doi.org/10.1016/j.ymthe.2017.03.029

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5474959/
https://www.ncbi.nlm.nih.gov/pubmed/28408179
http://dx.doi.org/10.1016/j.ymthe.2017.03.029

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