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.
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.
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