- April 23, 2021
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Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome.
Shao Y, Sztainberg Y, Wang Q, Bajikar SS, Trostle AJ, Wan YW, Jafar-Nejad P, Rigo F, Liu Z, Tang J, Zoghbi HY.
Sci Transl Med. 2021 Mar 3;13(583):eaaz7785.
Binding of phosphorothioate oligonucleotides with RNase H1 can cause conformational changes in the protein and alter the interactions of RNase H1 with other proteins
March 15th, 2021
Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)
February 16th, 2021