RNA-seq analysis reveals significant transcriptome changes in huntingtin-null human neuroblastoma cells.

ABSTRACT:

Background

Huntingtin (Htt) protein is the product of the gene mutated in Huntington's disease (HD), a fatal, autosomal dominant, neurodegenerative disorder. Normal Htt is essential for early embryogenesis and the development of the central nervous system. However, the role of Htt in adult tissues is less defined. Following the recent promising clinical trial in which both normal and mutant Htt mRNA were knocked down in HD patients, there is an urgent need to fully understand the molecular consequences of knocking out/down Htt in adult tissues. Htt has been identified as an important transcriptional regulator. Unbiased investigations of transcriptome changes with RNA-sequencing (RNA-Seq) have been done in multiple cell types in HD, further confirming that transcriptional dysregulat

SUBMITTER: Bensalel J 

PROVIDER: S-EPMC8252266 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature