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Aberrant splicing in Huntington's disease accompanies disrupted TDP-43 activity and altered m6A RNA modification.
Nguyen TB, Miramontes R, Chillon-Marinas C, Maimon R, Vazquez-Sanchez S, Lau AL, McClure NR, Wu Z, Wang KQ, England WE, Singha M, Stocksdale JT, Heath M, Jang KH, Jung S, Ling K, Jafar-Nejad P, McKnight JI, Ho LN, Dalahmah OA, Faull RLM, Steffan JS, Reidling JC, Jang C, Lee G, Cleveland DW, Lagier-Tourenne C, Spitale RC, Thompson LM. Nguyen TB, et al. Among authors: jafar nejad p. Nat Neurosci. 2025 Jan 6. doi: 10.1038/s41593-024-01850-w. Online ahead of print. Nat Neurosci. 2025. PMID: 39762660
Reduction of RAD23A extends lifespan and mitigates pathology in TDP-43 mice.
Guo X, Prajapati R, Chun J, Byun I, Gebis KK, Wang YZ, Ling K, Dalton C, Blair JA, Hamidianjahromi A, Bachmann G, Rigo F, Jafar-Nejad P, Savas JN, Lee MJ, Sreedharan J, Kalb RG. Guo X, et al. Among authors: jafar nejad p. bioRxiv [Preprint]. 2024 Sep 14:2024.09.10.612226. doi: 10.1101/2024.09.10.612226. bioRxiv. 2024. PMID: 39314471 Free PMC article. Preprint.
Modeling antisense oligonucleotide therapy in MECP2 duplication syndrome human iPSC-derived neurons reveals gene expression programs responsive to MeCP2 levels.
Bajikar SS, Sztainberg Y, Trostle AJ, Tirumala HP, Wan YW, Harrop CL, Bengtsson JD, Carvalho CMB, Pehlivan D, Suter B, Neul JL, Liu Z, Jafar-Nejad P, Rigo F, Zoghbi HY. Bajikar SS, et al. Among authors: jafar nejad p. Hum Mol Genet. 2024 Nov 8;33(22):1986-2001. doi: 10.1093/hmg/ddae135. Hum Mol Genet. 2024. PMID: 39277796 Free PMC article.
Targeting mechanistic target of rapamycin complex 2 attenuates immunopathology in Systemic Lupus Erythematosus.
Ai M, Zhou X, Carrer M, Jafar-Nejad P, Li Y, Gades N, Alexander M, Bautista MA, Garcia AAD, Zeng H. Ai M, et al. Among authors: jafar nejad p. bioRxiv [Preprint]. 2024 Aug 4:2024.08.01.606069. doi: 10.1101/2024.08.01.606069. bioRxiv. 2024. Update in: Rheumatology (Oxford). 2024 Dec 04:keae662. doi: 10.1093/rheumatology/keae662 PMID: 39131369 Free PMC article. Updated. Preprint.
Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study.
van den Berg LH, Rothstein JD, Shaw PJ, Babu S, Benatar M, Bucelli RC, Genge A, Glass JD, Hardiman O, Libri V, Mobach T, Oskarsson B, Pattee GL, Ravits J, Shaw CE, Weber M, Zinman L, Jafar-Nejad P, Rigo F, Lin L, Ferguson TA, Gotter AL, Graham D, Monine M, Inra J, Sinks S, Eraly S, Garafalo S, Fradette S. van den Berg LH, et al. Among authors: jafar nejad p. Lancet Neurol. 2024 Sep;23(9):901-912. doi: 10.1016/S1474-4422(24)00216-3. Epub 2024 Jul 23. Lancet Neurol. 2024. PMID: 39059407 Clinical Trial.
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