Gene Therapy for Inherited Metabolic Diseases

Over the last two decades, gene therapy has been successfully translated to many rare diseases. The number of clinical trials is rapidly expanding and some gene therapy products have now received market authorisation in the western world. Inherited metabolic diseases (IMD) are orphan diseases freque...

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Detalles Bibliográficos
Autores principales: Yilmaz, Berna Seker, Gurung, Sonam, Perocheau, Dany, Counsell, John, Baruteau, Julien
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Sciendo 2020
Materias:
Review Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518100/
https://www.ncbi.nlm.nih.gov/pubmed/33554501
http://dx.doi.org/10.34763/jmotherandchild.20202402si.2004.000009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518100/
https://www.ncbi.nlm.nih.gov/pubmed/33554501
http://dx.doi.org/10.34763/jmotherandchild.20202402si.2004.000009

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