1. University of Puerto Rico at Cayey
Written Proposal: Is there a way to slow down the Huntington’s disease?
Alejandra González Torres
Biol- 3009
Prof. Edgar Llera Santos

2. With this investigation I intend to slow down the Huntington’s disease. This is a
hereditary disease, and it affects 1 in 10,000 people in the United States. With this in
mind, I will use the huntingtin protein to find a way to slow down the production of the
polypeptide. The methodology consists of three phases, in which I will alter the DNA
sequence. The first phase is to use living cells, in the second phase I will use rats and
the last phase humans.
Huntington’s disease is a disorder passed through families, where the nerve cells
waste away or degenerate in the brain. It is caused by a genetic defect in chromosome
4. Huntington’s targets the brain, triggering the death of cells vital for movement, mood,
speech and memory. This disease affects 1 in 10,000 people in the United States. By
this I intend that as I study the huntingtin protein sequence I would be able to slow down
the Huntington’s disease.
The process I am going to use is based in three phases. The first phase is to use
the cells infected with the disease. As the cells express the gene, I will alter the
huntingtin protein in order to find the way to slow it down. After phase one is completed,
I will test it on rats. The rats need to be in mating stage, so I can also study how it
passes through the offspring. The control or negative group is composed by a minimum
of 10 couples of rats. The control group will only be infected with the disease. The
positive or experimental group will be composed of a minimum of 15 couples of rats,
they will also have the disease but this will be treated by changing the protein. As phase
two is completed, I will move on to phase 3 which is using humans. All of the subjects
will have the disease, but for this experiment the symptoms need to be in their first
appearance or that the subject doesn’t present the symptoms at all. The reason of this
is that it will be harder to study the disease with people that have advanced
Huntington’s disease. The control group will consist of 20 couples with the disease, and
the experimental group will consist of 30 couples. I would be treating the subjects with
the same procedure as the rats, if something goes wrong with the humans, I will review
what I am administrating and make the pertinent changes. There is no reason why I
chose the numbers but the more subjects I have the better number of variables I’m

3. going to have. In phase two and three, I would have the pertinent documents of the IRB
to ensure the security of the experiment and that everything will be controlled.
As I finalize each of the phases I expect that by altering the huntingtin protein it
can slow down the disease and later on give a head start to find a way to stop the
disease. In phase one; I expect that as I alter the protein it will slow the repetition of the
triplet. Phase two, I expect an improvement in the rats, and that as they are treated their
offspring will have the modified gene. The last phase, which is the third one, I expect the
same or better results as in the second phase.
References:
Georgia Tech [Internet] Atlanta (GA): Available: http://gtresearchnews.gatech.edu/huntingtons-
disease/
PubMed [Internet] Boston (MA): NeuroRx [cited 2004 April 1] Available:
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC534940/