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172 results

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Page 1
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
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Yoon Low RL, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G. Logan T, et al. Among authors: watts rj. Cell. 2024 Mar 14;187(6):1565-1566. doi: 10.1016/j.cell.2024.02.015. Cell. 2024. PMID: 38490183 Free article. No abstract available.
Author Correction: CD98hc is a target for brain delivery of biotherapeutics.
Chew KS, Wells RC, Moshkforoush A, Chan D, Lechtenberg KJ, Tran HL, Chow J, Kim DJ, Robles-Colmenares Y, Srivastava DB, Tong RK, Tong M, Xa K, Yang A, Zhou Y, Akkapeddi P, Annamalai L, Bajc K, Blanchette M, Cherf GM, Earr TK, Gill A, Huynh D, Joy D, Knight KN, Lac D, Leung AW, Lexa KW, Liau NPD, Becerra I, Malfavon M, McInnes J, Nguyen HN, Lozano EI, Pizzo ME, Roche E, Sacayon P, Calvert MEK, Daneman R, Dennis MS, Duque J, Gadkar K, Lewcock JW, Mahon CS, Meisner R, Solanoy H, Thorne RG, Watts RJ, Zuchero YJY, Kariolis MS. Chew KS, et al. Among authors: watts rj. Nat Commun. 2023 Sep 7;14(1):5516. doi: 10.1038/s41467-023-41355-x. Nat Commun. 2023. PMID: 37679403 Free PMC article. No abstract available.
CD98hc is a target for brain delivery of biotherapeutics.
Chew KS, Wells RC, Moshkforoush A, Chan D, Lechtenberg KJ, Tran HL, Chow J, Kim DJ, Robles-Colmenares Y, Srivastava DB, Tong RK, Tong M, Xa K, Yang A, Zhou Y, Akkapeddi P, Annamalai L, Bajc K, Blanchette M, Cherf GM, Earr TK, Gill A, Huynh D, Joy D, Knight KN, Lac D, Leung AW, Lexa KW, Liau NPD, Becerra I, Malfavon M, McInnes J, Nguyen HN, Lozano EI, Pizzo ME, Roche E, Sacayon P, Calvert MEK, Daneman R, Dennis MS, Duque J, Gadkar K, Lewcock JW, Mahon CS, Meisner R, Solanoy H, Thorne RG, Watts RJ, Zuchero YJY, Kariolis MS. Chew KS, et al. Among authors: watts rj. Nat Commun. 2023 Aug 19;14(1):5053. doi: 10.1038/s41467-023-40681-4. Nat Commun. 2023. PMID: 37598178 Free PMC article.
A TREM2-activating antibody with a blood-brain barrier transport vehicle enhances microglial metabolism in Alzheimer's disease models.
van Lengerich B, Zhan L, Xia D, Chan D, Joy D, Park JI, Tatarakis D, Calvert M, Hummel S, Lianoglou S, Pizzo ME, Prorok R, Thomsen E, Bartos LM, Beumers P, Capell A, Davis SS, de Weerd L, Dugas JC, Duque J, Earr T, Gadkar K, Giese T, Gill A, Gnörich J, Ha C, Kannuswamy M, Kim DJ, Kunte ST, Kunze LH, Lac D, Lechtenberg K, Leung AW, Liang CC, Lopez I, McQuade P, Modi A, Torres VO, Nguyen HN, Pesämaa I, Propson N, Reich M, Robles-Colmenares Y, Schlepckow K, Slemann L, Solanoy H, Suh JH, Thorne RG, Vieira C, Wind-Mark K, Xiong K, Zuchero YJY, Diaz D, Dennis MS, Huang F, Scearce-Levie K, Watts RJ, Haass C, Lewcock JW, Di Paolo G, Brendel M, Sanchez PE, Monroe KM. van Lengerich B, et al. Among authors: watts rj. Nat Neurosci. 2023 Mar;26(3):416-429. doi: 10.1038/s41593-022-01240-0. Epub 2023 Jan 12. Nat Neurosci. 2023. PMID: 36635496 Free PMC article.
Molecular architecture determines brain delivery of a transferrin receptor-targeted lysosomal enzyme.
Arguello A, Mahon CS, Calvert MEK, Chan D, Dugas JC, Pizzo ME, Thomsen ER, Chau R, Damo LA, Duque J, Fang M, Giese T, Kim DJ, Liang N, Nguyen HN, Solanoy H, Tsogtbaatar B, Ullman JC, Wang J, Dennis MS, Diaz D, Gunasekaran K, Henne KR, Lewcock JW, Sanchez PE, Troyer MD, Harris JM, Scearce-Levie K, Shan L, Watts RJ, Thorne RG, Henry AG, Kariolis MS. Arguello A, et al. Among authors: watts rj. J Exp Med. 2022 Mar 7;219(3):e20211057. doi: 10.1084/jem.20211057. Epub 2022 Feb 28. J Exp Med. 2022. PMID: 35226042 Free PMC article.
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Low RLY, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G. Logan T, et al. Among authors: watts rj. Cell. 2021 Sep 2;184(18):4651-4668.e25. doi: 10.1016/j.cell.2021.08.002. Epub 2021 Aug 26. Cell. 2021. PMID: 34450028 Free PMC article.
172 results