Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

• Main Authors: Jana Miniarikova (Author), Ilaria Zanella (Author), Angelina Huseinovic (Author), Tom van der Zon (Author), Evelyn Hanemaaijer (Author), Raygene Martier (Author), Annemart Koornneef (Author), Amber L Southwell (Author), Michael R Hayden (Author), Sander J van Deventer (Author), Harald Petry (Author), Pavlina Konstantinova (Author)
• Formato: Livro
• Publicado em: Elsevier, 2016-01-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Huntington's disease (HD) is a neurodegenerative disorder caused by accumulation of CAG expansions in the huntingtin (HTT) gene. Hence, decreasing the expression of mutated HTT (mtHTT) is the most upstream approach for treatment of HD. We have developed HTT gene-silencing approaches based on ex...