SIRNA THERAPEUTICS
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siRNA therapeutics: a clinical reality – Saw PE et. al
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they discovered that the silencing effectors in Caenorhabditis elegans (roundworm) were double
stranded RNAs. B
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This was proved by Single stranded (sense or antisense) RNA or double-stranded RNA encoding for
a muscle protein was injected into C. elegans. Both single-stranded RNA had no effect when
injected separately, but started to twitch when treated with double-stranded RNA, showing similar
phenotypes to C. elegans with defective muscle proteins. This indication denoted that the gene
coding for normal muscle movement had been silenced by the dsRNA.
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Why can this be useful in therapeutics?
On August 10, 2018, fda approved treatment of Hereditary Transthyretin Amyloidosis (hATTR). a
rare, progressive, and fatal disease.
Alnylam concluded that Patisiran is capable of significantly improving multiple clinical
manifestations of
hereditary transthyretin amyloidosis (Adams et al., 2018).
C19324413 Ross Quigley
C19368673 Naomi Ogbulafor