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Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.
Moazami MP, Rembetsy-Brown JM, Sarli SL, McEachern HR, Wang F, Ohara M, Wagh A, Kelly K, Krishnamurthy PM, Weiss A, Marosfoi M, King RM, Motwani M, Gray-Edwards H, Fitzgerald KA, Brown RH, Watts JK. Moazami MP, et al. Among authors: brown rh. Mol Ther. 2024 Oct 28:S1525-0016(24)00684-1. doi: 10.1016/j.ymthe.2024.10.024. Online ahead of print. Mol Ther. 2024. PMID: 39460376 Free article.
Gene therapy for neurologic disorders.
Eichler FS, Brown RH Jr. Eichler FS, et al. Among authors: brown rh jr. Neurotherapeutics. 2024 Jul;21(4):e00453. doi: 10.1016/j.neurot.2024.e00453. Neurotherapeutics. 2024. PMID: 39318035 Free PMC article. No abstract available.
Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs.
Gilbert JW, Kennedy Z, Godinho BMDC, Summers A, Weiss A, Echeverria D, Bramato B, McHugh N, Cooper D, Yamada K, Hassler M, Tran H, Gao FB, Brown RH Jr, Khvorova A. Gilbert JW, et al. Among authors: brown rh jr. Mol Ther Nucleic Acids. 2024 Jul 31;35(3):102291. doi: 10.1016/j.omtn.2024.102291. eCollection 2024 Sep 10. Mol Ther Nucleic Acids. 2024. PMID: 39233852 Free PMC article.
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy.
Rivera Flores IV, Monopoli K, Jackson S, Echeverria D, O'Reilly D, Brown RH, Khvorova A. Rivera Flores IV, et al. Among authors: brown rh. Nucleic Acid Ther. 2024 Oct;34(5):234-244. doi: 10.1089/nat.2024.0030. Epub 2024 Aug 27. Nucleic Acid Ther. 2024. PMID: 39189114
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.
Weiss A, Gilbert JW, Flores IVR, Belgrad J, Ferguson C, Dogan EO, Wightman N, Mocarski K, Echeverria D, Summers A, Bramato B, McHugh N, Furgal R, Yamada N, Cooper D, Monopoli K, Godinho BMDC, Hassler MR, Yamada K, Greer P, Henninger N, Brown RH Jr, Khvorova A. Weiss A, et al. Among authors: brown rh jr. bioRxiv [Preprint]. 2024 Jun 25:2024.06.20.599943. doi: 10.1101/2024.06.20.599943. bioRxiv. 2024. PMID: 38979291 Free PMC article. Preprint.
940 results