Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman Syndrome phenotype in mice.

Molecular therapy : the journal of the American Society of Gene Therapy

Clarke MT, Remesal L, Lentz L, Tan DJ, Young D, Thapa S, Namduri S, Borges B, Kirn G, Valencia J, Lopez ME, Lu JH, Shiow LR, Dindot S, Villeda S, Sanders SJ, MacKenzie TC. Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman Syndrome phenotype in mice. Mol Ther. 2024 Feb 06.

2024

https://researcherprofiles.org/profile/479745092

38327047

Clarke MT, Remesal L, Lentz L, Tan DJ, Young D, Thapa S, Namduri S, Borges B, Kirn G, Valencia J, Lopez ME, Lu JH, Shiow LR, Dindot S, Villeda S, Sanders SJ, MacKenzie TC

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Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman Syndrome phenotype in mice.