\nhttps:\/\/pubmed.ncbi.nlm.nih.gov\/31059641\/\n<\/div><\/figure>\n\n\n\n
Summary:<\/p>\n\n\n\n
Huntington’s disease is a genetic disorder that affects the brain, leading to problems with movement, thinking, and behavior. It occurs because of a mutation in the gene that makes a protein called huntingtin, causing this protein to build up in the brain and damage nerve cells. There is currently no cure for HD, but researchers are trying new treatments to help slow down the progression of the disease.<\/p>\n\n\n\n
This study tested a drug called IONIS-HTTRx (also known as tominersen) to see if it could lower the amount of harmful huntingtin protein in patients. The drug works by targeting the messenger RNA that tells cells to make the huntingtin protein, blocking the process so that less protein is produced by the cell.<\/p>\n\n\n\n
The researchers conducted a clinical trial, which is a study done on a small group of patients to check if the drug is safe and effective. A total of 46 patients with early Huntington’s disease participated in the trial. The patients were given different doses of the drug through spinal injections over three months.<\/p>\n\n\n\n
The results showed that the drug was successful in lowering the levels of the harmful huntingtin protein in the spinal fluid. In addition, most patients did not experience serious side effects, though some reported mild discomforts such as headaches and back pain from the injections.<\/p>\n\n\n\n
The researchers concluded that IONIS-HTTRx is a promising treatment for Huntington\u2019s disease because it can reduce the levels of the harmful protein. However, more studies are needed to see if lowering huntingtin protein can actually slow down the symptoms of the disease over a longer period of time.<\/p>\n\n\n\n
References<\/p>\n\n\n\n
Tabrizi, Sarah J et al. Targeting Huntingtin Expression in Patients with Huntington’s Disease.<\/p>\n\n\n\n
The New England journal of medicine vol. 380,24; <\/p>\n\n\n\n
https:\/\/doi.org\/10.1056\/NEJMoa1900907<\/a> (2019)<\/p>\n","protected":false},"excerpt":{"rendered":"Article: Targeting Huntingtin Expression in Patients with Huntington\u2019s Disease Summary: Huntington’s disease is a genetic disorder that affects the brain, leading to problems with movement, thinking, and behavior. It occurs because of a mutation in the gene that makes a protein called huntingtin, causing this protein to build up in the brain and damage nerve… <\/p>\n
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