Hi guys. we meet again after a long break. I know you guys miss me right? Because i miss you guys too!!. 

Ok now lets move on to our next topic. Can you guess what it is about? Hurm?..

.......................................................................................................................

Yeah correct.!! It is about promising research on Huntington Disease.

Ohh before we start, I'm so sad to announce that this will be our last topic that we discuss 

on Huntington Disease for this blog😢😢😢. Hope you guys will not dissapoint me by 

skipping this contents..

Now lets start on our topic.

According to the research done by the researchers from the University of British Columbia (UBC) and their colleagues on June 13, 2019, they have demonstrated for the first time that the drug, IONIS-HTTRX now known as RG6042 hereafter referred to as HTTRx successfully lowered levels of the mutant  huntingtin (mHTT) protein. HTTRx is a chemically modified synthetic oligomer that is perfectly complementary to a 20-nucleotide stretch of HTT mRNA. HTTRx binds to HTT mRNA by means of Watson–Crick base pairing, with hybridization resulting in endogenous RNase H1-mediated degradation of the HTT mRNA, thus inhibiting translation of the huntingtin protein.

Wait !! But, what is mutant huntington (mHTT) protein?

Mutant  huntington (mHTT) protein is the toxic protein that causes Huntington's disease in the central nervous system of the patients.

During the research, the trial enrolled 46 patients with early HD at nine study centres in Canada, the United Kingdom, and Germany. Of the 46 patients, 34 were randomized to receive the drug and 12 were randomized to receive placebo. Each participant received  HTTRx or placebo as a bolus intrathecal administration every 4 weeks for four doses and all study participants completed the study and have continued to receive the active drug in an ongoing open-label study.

The drug was administered monthly to patients via an injection directly into the cerebrospinal fluid. A cerebrospinal fluid (CSF) sample was obtained before each administration of HTTRx or placebo and either 4 or 8 weeks after the last dose was administered. The researchers, led by Dr. Sarah Tabrizi, director of the Huntington Disease Centre at University College London and global chief investigator of the IONIS-HTTRX clinical trial, found that the drug produced significant decreases in the levels of mutant huntington protein in the patients’ cerebrospinal fluid. No study participants experienced serious adverse reactions, suggesting that the treatment is safe and well tolerated by patients. In conclusion, they found that the antisense oligonucleotide drug HTTRx reduced the concentration of mutant HTT in the CSF of persons with Huntington’s disease. More generally, they found antisense-mediated protein suppression in the central nervous system of patients with a neurodegenerative disease.

The drug is currently being evaluated in a large, phase-three multi-centre clinical trial being performed at the Centre for Huntington Disease at UBC and other HD centres around the world. This study is designed to determine whether the treatment slows or stop the progression of the disease symptoms.

Ok guys, thats all for this time. I hope you guys can understand my explanation. If you guys want to
know detail about this research, just click link below

References : https://www.nejm.org/doi/10.1056/NEJMoa1900907