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      • Rare Disease Briefing Document
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        • Abstracts
        • Sponsors & Exhibitors
      • 2018 Annual Meeting
        • 2018 Meeting Overview
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        • 2018 Award Winners
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        • 2018 Video Gallery
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        • 2017 Meeting Overview
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        • 2017 Meeting Videos
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Taking oligonucleotide research to its full therapeutic potential

Author Archives: OTS

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  2. Article author OTS

Engineered anti-CRISPR proteins for optogenetic control of CRISPR-Cas9

Gene editingBy OTSNovember 30, 2018Leave a comment

Engineered anti-CRISPR proteins for optogenetic control of CRISPR-Cas9 …………… Bubeck F, Hoffmann MD, Harteveld Z, Aschenbrenner S, Bietz A, Waldhauer MC, Börner K, Fakhiri J, Schmelas C, Dietz L, Grimm D, Correia BE, Eils R, Niopek D. Nat Methods. 2018 Nov;15(11):924-927. PubMed 00

Genome-wide screening for functional long noncoding RNAs in human cells by Cas9 targeting of splice sites

Gene editing, Long noncoding RNABy OTSNovember 30, 2018Leave a comment

Genome-wide screening for functional long noncoding RNAs in human cells by Cas9 targeting of splice sites …………… Liu Y, Cao Z, Wang Y, Guo Y, Xu P, Yuan P, Liu Z, He Y, Wei W. Nat Biotechnol. 2018 Nov 5. PubMed 00

Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos

Gene editingBy OTSNovember 30, 2018Leave a comment

Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos …………… Zeng Y, Li J, Li G, Huang S, Yu W, Zhang Y, Chen D, Chen J, Liu J, Huang X. Mol Ther. 2018 Nov 7;26(11):2631-2637. PubMed 00

Predictable and precise template-free CRISPR editing of pathogenic variants

Gene editingBy OTSNovember 30, 2018Leave a comment

Predictable and precise template-free CRISPR editing of pathogenic variants …………… Shen MW, Arbab M, Hsu JY, Worstell D, Culbertson SJ, Krabbe O, Cassa CA, Liu DR, Gifford DK, Sherwood RI. Nature. 2018 Nov;563(7733):646-651. PubMed 00

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