Lipoprotein(a) Reduction in Persons with Cardiovascular Disease.
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Systemic Brain Delivery of Antisense Oligonucleotides across the Blood-Brain Barrier with a Glucose-Installed Polymeric Nanocarrier.
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Scn8a antisense oligonucleotide is protective in mouse models of SCN8A Encephalopathy and Dravet Syndrome.
Lenk GM, Jafar-Nejad P, Hill SF, Huffman LD, Smolen CE, Wagnon JL, Petit H, Yu W, Ziobro J, Bhatia K, Parent J, Giger RJ, Rigo F, Meisler MH. Ann Neurol. 2020 Jan 14. PubMed
Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides.
Østergaard ME, De Hoyos CL, Wan WB, Shen W, Low A, Berdeja A, Vasquez G, Murray S, Migawa MT, Liang XH, Swayze EE, Crooke ST, Seth PP. Nucleic Acids Res. 2020 Jan 25. PubMed
Antisense-Mediated Transcript Knockdown Triggers Premature Transcription Termination.
Lee JS, Mendell JT. Mol Cell. 2020 Jan 2. pii: S1097-2765(19)30924-4. PubMed
Directed RNase H Cleavage of Nascent Transcripts Causes Transcription Termination.
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A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo.
Zhong G, Wang H, He W, Li Y, Mou H, Tickner ZJ, Tran MH, Ou T, Yin Y, Diao H, Farzan M. Nat Biotechnol. 2019 Dec 23. PubMed
Knockdown of GADD34 in neonatal mutant SOD1 mice ameliorates ALS.
Ghadge GD, Sonobe Y, Camarena A, Drigotas C, Rigo F, Ling KK, Roos RP. Neurobiol Dis. 2019 Dec 16;136:104702. PubMed
Targeting Repeated Regions Unique to a Gene Is an Effective Strategy for Discovering Potent and Efficacious Antisense Oligonucleotides.
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Golgi-endosome transport mediated by M6PR facilitates release of antisense oligonucleotides from endosomes
Liang XH, Sun H, Hsu CW, Nichols JG, Vickers TA, De Hoyos CL, Crooke ST. Nucleic Acids Res. 2019 Dec 16. pii: gkz1171. PubMed
