Efficacy of Postnatal In Vivo Nonsense Suppression Therapy in a Pax6 Mouse Model of Aniridia

Nonsense mutations leading to premature stop codons are common occurring in approximately 12% of all human genetic diseases. Thus, pharmacological nonsense mutation suppression strategies would be beneficial to a large number of patients if the drugs could be targeted to the affected tissues at the...

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Detalles Bibliográficos
Autores principales: Wang, Xia, Gregory-Evans, Kevin, Wasan, Kishor M., Sivak, Olena, Shan, Xianghong, Gregory-Evans, Cheryl Y.
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/PMC5440746/
https://www.ncbi.nlm.nih.gov/pubmed/28624217
http://dx.doi.org/10.1016/j.omtn.2017.05.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5440746/
https://www.ncbi.nlm.nih.gov/pubmed/28624217
http://dx.doi.org/10.1016/j.omtn.2017.05.002

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