Preclinical Testing in Translational Animal Models of Prader-Willi Syndrome: Overview and Gap Analysis

Prader-Willi syndrome (PWS) is a rare neurodevelopmental disorder causing endocrine, musculoskeletal, and neurological dysfunction. PWS is caused by the inactivation of contiguous genes, complicating the development of targeted therapeutics. Clinical trials are now underway in PWS, with more trials...

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Detalles Bibliográficos
Autores principales: Carias, K. Vanessa, Wevrick, Rachel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6447752/
https://www.ncbi.nlm.nih.gov/pubmed/30989085
http://dx.doi.org/10.1016/j.omtm.2019.03.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6447752/
https://www.ncbi.nlm.nih.gov/pubmed/30989085
http://dx.doi.org/10.1016/j.omtm.2019.03.001

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