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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...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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American Society of Gene & Cell Therapy
2019
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| 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 |