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Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa
By combining next generation whole exome sequencing and induced pluripotent stem cell (iPSC) technology we found that an Alu repeat inserted in exon 9 of the MAK gene results in a loss of normal MAK transcript and development of human autosomal recessive retinitis pigmentosa (RP). Although a relativ...
Autores principales: | Tucker, Budd A., Burnight, Erin R., Cranston, Cathryn M., Ulferts, Mallory J., Luse, Meagan A., Westfall, Trudi, Scott, C. Anthony, Marsden, Autumn, Gibson-Corley, Katherine, Wiley, Luke A., Han, Ian C., Slusarski, Diane C., Mullins, Robert F., Stone, Edwin M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9159943/ https://www.ncbi.nlm.nih.gov/pubmed/34518651 http://dx.doi.org/10.1038/s41434-021-00291-5 |
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