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Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Clarke MT, Remesal L, Lentz L, Tan DJ, Young D, Thapa S, Namuduri SR, Borges B, Kirn G, Valencia J, Lopez ME, Lui JH, Shiow LR, Dindot S, Villeda S, Sanders SJ, MacKenzie TC. Clarke MT, et al. Among authors: remesal l. Mol Ther. 2024 Apr 3;32(4):935-951. doi: 10.1016/j.ymthe.2024.02.004. Epub 2024 Feb 6. Mol Ther. 2024. PMID: 38327047 Free PMC article.
PBX1 acts as terminal selector for olfactory bulb dopaminergic neurons.
Remesal L, Roger-Baynat I, Chirivella L, Maicas M, Brocal-Ruiz R, Pérez-Villalba A, Cucarella C, Casado M, Flames N. Remesal L, et al. Development. 2020 Apr 13;147(8):dev186841. doi: 10.1242/dev.186841. Development. 2020. PMID: 32156753 Free article.