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Progress and prospects of gene therapy clinical trials for the muscular dystrophies

Clinical trials represent a critical avenue for new treatment development, where early phases (I, I/II) are designed to test safety and effectiveness of new therapeutics or diagnostic indicators. A number of recent advances have spurred renewed optimism toward initiating clinical trials and developi...

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Detalles Bibliográficos
Publicado en:Hum Mol Genet
Autores principales: Bengtsson, Niclas E., Seto, Jane T., Hall, John K., Chamberlain, Jeffrey S., Odom, Guy L.
Formato: Artigo
Lenguaje:Inglês
Publicado: Oxford University Press 2016
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4802376/
https://ncbi.nlm.nih.gov/pubmed/26450518
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv420
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