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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...

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Detalles Bibliográficos
Autores principales:	Braga, Luiza Amara Maciel, Conte Filho, Carlos Gilbert, Mota, Fabio Batista
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	SAGE Publications 2022
Materias:	Original Research
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

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