GENE TARGET: A framework for evaluating Mendelian neurodevelopmental disorders for gene therapy

Interest in gene-based therapies for neurodevelopmental disorders is increasing exponentially, driven by the rise in recognition of underlying genetic etiology, progress in genomic technology, and recent proof of concept in several disorders. The current prioritization of one genetic disorder over a...

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Detalles Bibliográficos
Autores principales: Chopra, Maya, Modi, Meera E., Dies, Kira A., Chamberlin, Nancy L., Buttermore, Elizabeth D., Brewster, Stephanie Jo, Prock, Lisa, Sahin, Mustafa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9478871/
https://www.ncbi.nlm.nih.gov/pubmed/36156879
http://dx.doi.org/10.1016/j.omtm.2022.08.007

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9478871/
https://www.ncbi.nlm.nih.gov/pubmed/36156879
http://dx.doi.org/10.1016/j.omtm.2022.08.007

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