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Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency

The pathway to a clinical gene therapy product often involves many changes of course and strategy before obtaining successful results. Here we outline the methodologies, both clinical and preclinical, that went into developing a gene therapy approach to the treatment of alpha-1 antitrypsin deficienc...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther Methods
Main Authors: Gruntman, Alisha M., Flotte, Terence R.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4559188/
https://ncbi.nlm.nih.gov/pubmed/26067712
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2015.086
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