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      • Rare Disease Briefing Document
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      • 2019 Annual Meeting
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        • Abstracts
        • Sponsors & Exhibitors
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        • 2018 Meeting Overview
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Taking oligonucleotide research to its full therapeutic potential

Author Archives: OTS

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Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction

Alternative splicing, Splice modulationBy OTSApril 30, 2020Leave a comment

Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction …………… Wang L, Kempton JB, Jiang H, Jodelka FM, Brigande AM, Dumont RA, Rigo F, Lentz JJ, Hastings ML, Brigande JV. Nucleic Acids Res. 2020 Apr 6. PubMed 00

Directed evolution of adenine base editors with increased activity and therapeutic application

Gene editingBy OTSApril 30, 2020Leave a comment

Directed evolution of adenine base editors with increased activity and therapeutic application …………… Gaudelli NM, Lam DK, Rees HA, Solá-Esteves NM, Barrera LA, Born DA, Edwards A, Gehrke JM, Lee SJ, Liquori AJ, Murray R, Packer MS, Rinaldi C, Slaymaker IM, Yen J, Young LE, Ciaramella G. Nat Biotechnol. 2020 Apr 13. PubMed 00

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

Gene editingBy OTSApril 30, 2020Leave a comment

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity …………… Yu Y, Leete TC, Born DA, Young L, Barrera LA, Lee SJ, Rees HA, Ciaramella G, Gaudelli NM. Nat Commun. 2020 Apr 28;11(1):2052. PubMed 00

Imaging small molecule-induced endosomal escape of siRNA

Ago/siRNA/saRNA, DeliveryBy OTSApril 30, 2020Leave a comment

Imaging small molecule-induced endosomal escape of siRNA …………… Du Rietz H, Hedlund H, Wilhelmson S, Nordenfelt P, Wittrup A. Nat Commun. 2020 Apr 14;11(1):1809. PubMed 00

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