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- W2912572612 abstract "The first published description of therapeutic applications of antisense oligonucleotide (ASO) technology occurred in the late 1970s and was followed by the founding of commercial companies focused on developing antisense therapeutics in the late 1980s. Since the late 1980s, there has been steady progress in improving the technology platform, taking advantage of advances in oligonucleotide chemistry and formulations as well as increased understanding of the distribution and safety of ASOs. There are several approved ASO drugs and a broad pipeline in development. In addition, advances in understanding human disease, including the genetic basis for most monogenic diseases and the availability of the full human genome sequence, have created numerous therapeutic applications for the technology. I summarize the state of the technology and highlight how advances in the technology position ASOs to be an important contributor to future medicines." @default.
- W2912572612 created "2019-02-21" @default.
- W2912572612 creator A5083296071 @default.
- W2912572612 date "2019-01-27" @default.
- W2912572612 modified "2023-10-11" @default.
- W2912572612 title "Therapeutic Antisense Oligonucleotides Are Coming of Age" @default.
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- W2912572612 doi "https://doi.org/10.1146/annurev-med-041217-010829" @default.
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- W2912572612 hasPublicationYear "2019" @default.
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