Rational Engineering and Preclinical Evaluation of Neddylation and SUMOylation Site Modified Adeno-Associated Virus Vectors in Murine Models of Hemophilia B and Leber Congenital Amaurosis

Synthetic engineering of viral vectors such as adeno-associated virus (AAV) is crucial to overcome host transduction barriers observed during clinical gene therapy. We reasoned that exploring the role of cellular ubiquitin-like modifiers (UBLs) such as Neddylation or SUMOylation during AAV transduct...

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Detalles Bibliográficos
Autores principales: Maurya, Shubham, Mary, Bertin, Jayandharan, Giridhara R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2019
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6919284/
https://www.ncbi.nlm.nih.gov/pubmed/31642343
http://dx.doi.org/10.1089/hum.2019.164

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6919284/
https://www.ncbi.nlm.nih.gov/pubmed/31642343
http://dx.doi.org/10.1089/hum.2019.164

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