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DNAzyme Cleavage of CAG Repeat RNA in Polyglutamine Diseases

CAG repeat expansion is the genetic cause of nine incurable polyglutamine (polyQ) diseases with neurodegenerative features. Silencing repeat RNA holds great therapeutic value. Here, we developed a repeat-based RNA-cleaving DNAzyme that catalyzes the destruction of expanded CAG repeat RNA of six poly...

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Detalles Bibliográficos
Autores principales: Zhang, Nan, Bewick, Brittani, Schultz, Jason, Tiwari, Anjana, Krencik, Robert, Zhang, Aijun, Adachi, Kaho, Xia, Guangbin, Yun, Kyuson, Sarkar, Partha, Ashizawa, Tetsuo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8609077/
https://www.ncbi.nlm.nih.gov/pubmed/34160773
http://dx.doi.org/10.1007/s13311-021-01075-w