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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...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2022
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| Materias: | |
| 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 |