One of the things about reading Nature Briefing: they always seem put you au fait with the cutting-edge medical techniques, and the science that drives them, a little way ahead of the mainstream. Which is why we are showcasing their story First for Gene Targeting RNA therapy, not just because it’s interesting in itself, but because it is a beautiful illustration of the progress in RNA based medicine, even since we started this humble little blog six years ago. First, here’s their snippet
An RNA therapy for rare forms of amyotrophic lateral sclerosis (ALS) has shown promising results in the first person to receive it. He experienced improved symptoms and continued to work as a physician a year after starting treatment. Antisense oligonucleotide therapy uses short strands of genetic material to target RNA produced by a faulty gene called CHCHD10. Roughly 5–10% of people with ALS have a known genetic mutation, and antisense oligonucleotide therapies could someday benefit them and others with neurodegenerative diseases caused by rare mutations.
Now, we admit CHCHD10 is a rare mutation, apparently occurring in less than one per cent of ALS cases. There are many other genes involved among the different types of ALS, and, who knows, maybe some epigenetic and environmental factors as well. But to raise that is to be like a man who has won the lottery complaining that the prize didn’t come gift wrapped in Harvey Nicholls wrapping paper. Because, gentle readers-this is a start and a magnificent one. Better still, it’s part of a much bigger story of RNA based medicine advancing on many fronts. Readers will recall how the sudden arrival of RNA vaccines suddenly lifted the world from its COVID-19 based Slough of Despond in 2021. But astute readers will recall that even before COVID, RNA‑based drugs were quietly succeeding: briefly, we will cite Patisiran , ansiRNA therapy for hereditary amyloidosis;Nusinersen — antisense therapy for spinal muscular atrophy; and Givosiran, and inclisiran — RNA drugs for metabolic and cardiovascular conditions; and then let you go off to do your own reading. Yet this in turn is but part of an even bigger picture, a new frontier if you like, of therapies which we hope might deliver RNA constructs to reduce toxic proteins in motor neurons, use antisense or siRNA to knock down mutant SOD1, C9orf72, or other ALS‑linked genes, and even exploit improved delivery systems (lipid nanoparticles, viral vectors) that can finally reach neural tissue. But time will answer many of these questions
And our takeaway? It’s our usual . Using science, and its simple but effective tools of evidence and reason will always make you feel better. So if you start tinkering with basic research and its funding, you do so at your peril.
#RNA #RNA therapy #ALS #medicine #health #COVID 19 # # CHCHD10 #gene therapy #neurodegenerative diseases
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