Huntington's disease is a genetic brain disorder that has a widespread effect on how our bodies function mutations in the huntington or HTT gene causes a progressive breakdown of nerve cells or neurons located in the brain this eventually affects an individual's ability to move their mood and even how they think the exact function of the HTT gene and healthy adults is not yet fully clear but as an adult the inherited mutation in the HTT gene leads to the production of toxic protein that causes the death of neurons in the brain this disease is devastating to the affected person and is extremely difficult for their family to as independence is slowly lost it is hard for caretakers to manage both their emotional and medical needs not to mention the stress related to the potential risk of the disease being passed along to their children this is a 50/50 chance however gene therapy might offer hope for this disease which currently has no cure or effective treatment options gene therapy typically changes how cells produce important proteins by replacing a gene that is missing or abnormal in the case of Huntington's gene therapy would actually help lower the cells production of the toxic HTT protein ideally slowing the breakdown of neurons in the brain the two main approaches currently in clinical trials use either micro RNAs or antisense oligonucleotides to accomplish this to talk more about micro RNA here's dr. beverly davidson microRNAs are short RNA molecules that prevent or reduce the production of a particular protein my Carney's work as a check and balance system to control protein levels by binding to and destroying a messenger RNA that would have produced that protein as an example we can reduce the level of toxic proteins that are causing Huntington's disease by using gene therapy to insert mic renée's to bind to an inhibit production of the mutant huntingtin protein the micro RNA needs to be delivered into a cell by a vector which is often derived from viruses Auri all viral genes are removed and the vector is modified to only deliver therapeutic genetic material the modified cells with the micro RNA provide instructions to produce much less HTT protein the other approach antisense oligonucleotide referred to as a SOS is an anti sense therapy that is designed to block or alter how cells express specific genes anti sense therapy uses synthetic DNA or RNA mimics that bind to the faulty HTT gene to block its ability to produce the harmful protein that causes the death of neurons a key difference between these two techniques is that antisense oligonucleotides typically require multiple doses while micro RNAs aim to be administered only one time researchers continue to work optimistically in clinical trials to safely and effectively slow down the progression of Huntington's disease for more information and resources about gene therapy for Huntington's disease go to ASG CT org you