Kennedys Disease Community
Developing an antisense therapy approach for Kennedy’s disease

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03-04-2020, 06:24 AM
Developing an antisense therapy approach for Kennedy’s disease
I entered the Kennedy Disease Association's Facebook and I have one document that is posted on February 2. Have you heard about this? Is that good news?
03-04-2020, 07:12 AM
Hi Jin, welcome to the forums.

I consider the work on ASOs for KD potentially good news. There have been more successes so far for other diseases using ASO approaches than for gene editing so the hurdles to making it to treatment are likely smaller.

But I'm personally not excited about it. Men with KD have only mutant AR and knocking it down with ASOs will likely aggravate symptoms of deficient androgen signaling encountered with androgen suppressing drugs. Perhaps they will be able to minimize this with tissue specific suppression but considering skeletal muscle appears to be a primary site of KD pathology and it is also an important tissue for andogen signaling this approach will be imperfect at best.

My view is biased by personal experience of having gone from very rapid deterioration to making gains in health, fitness, performance and body composition through lifestyle changes that have resulted in a huge increase in testosterone which should be making KD much worse but instead seems to be contributing to my efforts to lose fat and regain muscle. I enjoy feeling like a teenager again at 55 and would only consider taking a drug to suppress androgen signaling if I again start feeling diseased and desperate.