Targeted exon skipping with AAV-mediated split adenine base editors

Techniques for exclusion of exons from mature transcripts have been applied as gene therapies for treating many different diseases. Since exon skipping has been traditionally accomplished using technologies that have a transient effect, it is particularly important to develop new techniques that ena...

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Detalles Bibliográficos
Autores principales: Winter, Jackson, Luu, Alan, Gapinske, Michael, Manandhar, Sony, Shirguppe, Shraddha, Woods, Wendy S., Song, Jun S., Perez-Pinera, Pablo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6796986/
https://www.ncbi.nlm.nih.gov/pubmed/31636954
http://dx.doi.org/10.1038/s41421-019-0109-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6796986/
https://www.ncbi.nlm.nih.gov/pubmed/31636954
http://dx.doi.org/10.1038/s41421-019-0109-7

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