Sentences with phrase «start of the huntingtin»
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.
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2015 will see the start of the first human clinical trial of a gene silencing or huntingtin - lowering drug, which specifically aims to reduce production of mutant huntingtin in the brains of HD patients.
Normally, neurons start life deep within the developing brain, migrate out to the surface and then make a network of connections with others, but Sandrine Humbert's group showed that those without huntingtin get stuck, never making it to where they need to go.
The expanded CAG sequence in the HD gene results in a huntingtin protein with too many glutamines at the start of it.
There are many ways of achieving the goal of lowering mutant huntingtin, expected to start new trials soon or already in early trials.