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DNL343 is an investigational CNS penetrant eukaryotic initiation factor 2B activator that prevents and reverses the effects of neurodegeneration caused by the integrated stress response.
Yulyaningsih E, Suh JH, Fanok M, Chau R, Solanoy H, Takahashi R, Bakardjiev AI, Becerra I, Benitez NB, Chiu CL, Davis SS, Dowdle WE, Earr T, Estrada AA, Gill A, Ha C, Haddick PCG, Henne KR, Larhammar M, Leung AW, Maciuca R, Memarzadeh B, Nguyen HN, Nugent AA, Osipov M, Ran Y, Rebadulla K, Roche E, Sandmann T, Wang J, Lewcock JW, Scearce-Levie K, Kane LA, Sanchez PE. Yulyaningsih E, et al. Among authors: leung aw. Elife. 2024 Sep 17;12:RP92173. doi: 10.7554/eLife.92173. Elife. 2024. PMID: 39287504 Free PMC article.
Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4.
Sai H, Ollington B, Rezek FO, Chai N, Lane A, Georgiadis T, Bainbridge J, Michaelides M, Sacristan-Reviriego A, Perdigão PRL, Leung A, van der Spuy J. Sai H, et al. Among authors: leung a. Mol Ther Nucleic Acids. 2024 Feb 13;35(1):102148. doi: 10.1016/j.omtn.2024.102148. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2024. PMID: 38439910 Free PMC article.
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: leung aw. 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: leung aw. Nat Commun. 2023 Aug 19;14(1):5053. doi: 10.1038/s41467-023-40681-4. Nat Commun. 2023. PMID: 37598178 Free PMC article.
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