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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...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Nature Publishing Group UK
2019
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| Materias: | |
| 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 |