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I thought that people on this board might be interested in this nice write-up by Eric Minikel, a prion disease researcher: Huntingtin becomes the huntingted
Many of you probably already know about the antisense oligonucleotide (ASO) therapy for spinal muscular atrophy, which was approved in 2016. Now a press report suggests that a company has developed an ASO against Huntington's disease that is injected intrathecally but still leads to a significant decrease in the amount of huntingtin protein in the brain in a clinical trial.
This treatment would obviously be revolutionary if successful, and would emphasize the potential for ASOs in treating neurologic disease.
Many of you probably already know about the antisense oligonucleotide (ASO) therapy for spinal muscular atrophy, which was approved in 2016. Now a press report suggests that a company has developed an ASO against Huntington's disease that is injected intrathecally but still leads to a significant decrease in the amount of huntingtin protein in the brain in a clinical trial.
This treatment would obviously be revolutionary if successful, and would emphasize the potential for ASOs in treating neurologic disease.
