Isis Pharmaceuticals, a biotech company that specializes in RNA-targeting therapies, has recently had two major breakthroughs in neurological diseases (for their work in ALS see Jan 2013 news, Oct 2013 news). The company, in a partnership with Roche, has created an antisense oligonucleotide (ASO)-based therapy against Huntington’s disease (HD), called ISIS-HTTRx. Injected directly into spinal fluid, the drug will hopefully prevent or delay the progression of HD by inhibiting production of the aberrant HTT protein. Phase 1 of testing ISIS-HTTRx in humans is now underway at the University College London Hospital in the UK, and will continue in patients across Europe and Canada. Isis Pharma has also collaborated with scientists from The Jackson Laboratory in Maine, to develop a novel mouse model of spinal muscular atrophy (SMA), a pediatric motor neuron disease that leads to impaired mobility and muscle atrophy. The researchers were able to increase the lifespan and neuromuscular health of the mice with ASOs designed to increase levels of the survival motor neuron 1 (SMN1) protein. The results were published last month in PNAS.
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