piggyBac-mediated phenotypic correction of factor VIII deficiency

Hemophilia A, caused by a deficiency in factor VIII (FVIII), is the most severe inherited bleeding disorder. Hemophilia A is an attractive gene therapy candidate because even small increases in FVIII levels will positively alter the phenotype. While several vectors are under investigation, gene addi...

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Detalles Bibliográficos
Autores principales: Staber, Janice M, Pollpeter, Molly J, Arensdorf, Angela, Sinn, Patrick L, Rutkowski, D Thomas, McCray, Paul B
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362371/
https://www.ncbi.nlm.nih.gov/pubmed/26015980
http://dx.doi.org/10.1038/mtm.2014.42

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362371/
https://www.ncbi.nlm.nih.gov/pubmed/26015980
http://dx.doi.org/10.1038/mtm.2014.42

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