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Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?
INTRODUCTION: Rare genetic diseases affect millions of people worldwide. Most of them are caused by defective genes that impair quality of life and can lead to premature death. As genetic therapies aim to fix or replace defective genes, they are considered the most promising treatment for rare genet...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2022
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10032453/ https://www.ncbi.nlm.nih.gov/pubmed/37180410 http://dx.doi.org/10.1177/26330040221100840 |