A study in the New England Journal of Medicine has found that a new form of genetic therapy may help slow down the neurodegenerative effects of a disease called adrenoleukodystrophy (ALD). Researchers decided to use a “disabled form” of the HIV virus for proceeding with the treatment plan, which was considered quite risky at the time.
The plan of action consisted of the following: First, take a sample of bone marrow stem cells from a patient with a gene mutation heavily correlated with the ALD disease. Then, put in a desired gene in the bone marrow stem cells and put them back in the patient. The hope would be that the stem cells would enter the brain and become glial cells in order to stop further degeneration in the brain. The preferred timeline for all of this to occur was in about a year or so after putting the stem cells with the new genes back into the patient. The study showed that 15 out of the 17 participants no longer showed any symptoms after this form of treatment. This study is important because it shows promising outcomes to this type of therapy for perhaps other neurodegenerative diseases. I am excited as to what the future has to offer.
- Eyasu Kebede
Source: https://www.nytimes.com/2017/10/05/health/gene-therapy-brain-disease.html
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