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Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

In recent years, gene therapy has been raising hopes toward viable treatment strategies for rare genetic diseases for which there has been almost exclusively supportive treatment. We here review this progress at the pre-clinical and clinical trial levels as well as market approvals within diseases t...

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Detalles Bibliográficos
Autores principales: Jensen, Thomas Leth, Gøtzsche, Casper René, Woldbye, David P. D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8527017/
https://www.ncbi.nlm.nih.gov/pubmed/34690692
http://dx.doi.org/10.3389/fnmol.2021.695937

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