PARIS-htt: an optimised expression platform to study huntingtin reveals functional domains required for vesicular trafficking.

ABSTRACT:

Background

Huntingtin (htt) is a multi-domain protein of 350 kDa that is mutated in Huntington's disease (HD) but whose function is yet to be fully understood. This absence of information is due in part to the difficulty of manipulating large DNA fragments by using conventional molecular cloning techniques. Consequently, few studies have addressed the cellular function(s) of full-length htt and its dysfunction(s) associated with the disease.

Results

We describe a flexible synthetic vector encoding full-length htt called pARIS-htt (Adaptable, RNAi Insensitive &Synthetic). It includes synthetic cDNA coding for full-length human htt modified so that: 1) it is improved for codon usage, 2) it is insensitive to four different siRNAs allowing gene replacement studies, 3) it contain

SUBMITTER: Pardo R 

PROVIDER: S-EPMC2887845 | biostudies-literature | 2010 Jun

REPOSITORIES: biostudies-literature