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2017 ; 25
(5
): 1069-1075
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FDA-Approved Oligonucleotide Therapies in 2017
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Stein CA
; Castanotto D
Mol Ther
2017[May]; 25
(5
): 1069-1075
PMID28366767
show ga
Oligonucleotides (oligos) have been under clinical development for approximately
the past 30 years, beginning with antisense oligonucleotides (ASOs) and apatmers
and followed about 15 years ago by siRNAs. During that lengthy period of time,
numerous clinical trials have been performed and thousands of trial participants
accrued onto studies. Of all the molecules evaluated as of January 2017, the
regulatory authorities assessed that six provided clear clinical benefit in
rigorously controlled trials. The story of these six is given in this review.