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robbiep | 5 months ago

Huntingtons is not unique but certainly notable because it is caused by repeat sequences and therefore uniquely suited to mRNA silencing in this manner. There are very few other progressive (and I presume you also mean neurological) conditions, but also applied to the rest of the body, where this is the pathophysiology. For example, currently there is no immediate expansion of this to ie Parkinson’s (different pathophysiological basis) Lewy body (although maybe?) alzheimers (again possibly depending on whether it is tau, amyloid beta or simply ‘type 3 diabetes’) and nothing whatsoever for vascular dementia or ALS

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