First off, all of this new research on the role of the helper complex and mTOR in making
extra-long huntingtin protein was done in cells or mice.
During translation, however, the RNA instructions for
extra-long huntingtin can also interact with a helper complex (the assistant chef from our analogy).
The more we understand about how the normal and
extra-long huntingtin proteins are made and work in brain cells, the better equipped we'll be in the search for HD treatments.
Not exact matches
It's the
extra-long copy of the
huntingtin gene that makes neurons sick, because it causes them to produce an
extra-long, harmful version of the
huntingtin protein.
In HD, a repeated sequence of letters in this gene leads to an
extra-long form of
huntingtin protein that can wreak havoc in brain cells over long periods of time.
Since the helper complex mostly interacts with the extra-length
huntingtin instructions, interfering with it should reduce translation of the
extra-long variety.
Everyone has two copies of the
huntingtin gene but Huntington's disease is caused by a copy that's
extra-long.
If we could do it in people, it'd mean that
extra-long or «mutant»
huntingtin would never even have a chance to make neurons sick.
As expected, they found that the normal and
extra-long genetic instructions were both translated into
huntingtin proteins when they met up with a ribosome (the chef from our analogy above).