Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier.
Barker SJ, Thayer MB, Kim C, Tatarakis D, Simon MJ, Dial R, Nilewski L, Wells RC, Zhou Y, Afetian M, Akkapeddi P, Chappell A, Chew KS, Chow J, Clemens A, Discenza CB, Dugas JC, Dwyer C, Earr T, Ha C, Ho YS, Huynh D, Lozano EI, Jayaraman S, Kwan W, Mahon C, Pizzo M, Robles-Colmenares Y, Roche E, Sanders L, Stergioulis A, Tong R, Tran H, Zuchero Y, Estrada AA, Gadkar K, Koth CMM, Sanchez PE, Thorne RG, Watts RJ, Sandmann T, Kane LA, Rigo F, Dennis MS, Lewcock JW, DeVos SL.
Barker SJ, et al. Among authors: watts rj.
Sci Transl Med. 2024 Aug 14;16(760):eadi2245. doi: 10.1126/scitranslmed.adi2245. Epub 2024 Aug 14.
Sci Transl Med. 2024.
PMID: 39141703