Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …

Antisense oligonucleotides represent a novel therapeutic platform for the discovery of
medicines that have the potential to treat most neurodegenerative diseases. Antisense …

Huntington's disease is a neurodegenerative disorder caused by a polyglutamine repeat in
the Huntingtin gene (HTT). Although suppressing the expression of mutant HTT (mHTT) has …

There are no disease-modifying treatments for adult human neurodegenerative diseases.
Here we test RNA-targeted therapies in two mouse models of spinocerebellar ataxia type 2 …

The primary cause of Huntington's disease (HD) is expression of huntingtin with a
polyglutamine expansion. Despite an absence of consensus on the mechanism (s) of …

A comprehensive genetics-based precision medicine strategy to selectively and
permanently inactivate only mutant, not normal allele, could benefit many dominantly …

Huntington disease (HD) is a fatal progressive neurodegenerative disorder caused by an
inherited mutation in the huntingtin (HTT) gene, which encodes mutant HTT protein. Though …

Huntington disease is devastating to patients and their families—with autosomal dominant
inheritance, onset typically in the prime of adult life, progressive course, and a combination …

The majority of currently available drugs and tool compounds exhibit an inhibitory
mechanism of action and there is a relative lack of pharmaceutical agents that are capable …

There are few disease-modifying therapeutics for neurodegenerative diseases, but
successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal …