Introduction:

            My primary article is titled “Targeting Huntingtin Expression in Patients with Huntington’s Disease” The journal was created by a total of seventeen authors with the lead being Sarah J. Tabrizi. The authors published this article under the New England Journal of Medicine in 2019. 

Background: 

Huntington’s Disease is a dominant neurodegenerative disorder where cells progressively deteriorate, ultimately leading to cell death or dysfunction. It stems from a trinucleotide repeat of cytosine, adenine, and guanine (CAG) within a specific gene. While typically repeating 10 to 35 times, individuals with Huntington’s can exhibit repeats of 36 to 120 times. 

Methods: 

Those conducting the study decided to gather a group of adults who displayed signs of early Huntington’s disease. The adults were split into a 3:1 ratio where the majority received a dose of HTTRx. The smaller portion of the group received a placebo drug. The trial lasted a total of sixteen weeks, where another dose was given every four weeks. It is said that the dose selection went through a previous trial on mice and non-human primates before this one was conducted. 

Results: 

            All patients received doses in increasing increments and completed the trial all the way through. There were no serious adverse events seen however, no relevant adverse changes could be seen. 

Conclusion: 

The group tested their drug on a controlled population. The drug was injected near the spinal cord. It was found that the drug did not cause major side effects and, that it could reduce the level of harmful protein that is known to be linked with the disease. 

Citations

 Tabrizi J, Sarah. et al. Targeting Huntington Expression in Patients with Huntington’s
Disease. New England Journal of Medicine. doi: 10.1056/NEJMoa1900907. (2019)

Bethesda, MD. Huntington’s disease. 7.1.20