After an exciting day of science yesterday, day 2 saw updates on strategies to rid cells of
the harmful mutant huntingtin protein and exciting reports on current and planned clinical trials.
Designing drugs that tell our cells to make less of
the harmful mutant huntingtin protein is one of the most promising approaches to treating Huntington's disease.
Not exact matches
The study successfully countered
harmful effects of
mutant huntingtin and protected nerve cells in several models of Huntington's disease.
The zinc finger protein works by targeting the
mutant copies of the
Huntingtin gene, repressing its ability to express and create
harmful proteins.
R6 / 2 mice have been genetically altered with an abnormal gene that makes them produce a
harmful fragment of the
mutant huntingtin protein
When a person has too many CAGs in a row near the start of the
huntingtin gene, a
harmful «
mutant» form of the protein is produced based on the instructions in the gene.
That causes cells to manufacture a
harmful protein —
mutant huntingtin.