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Page 1
Approaches to Gene Modulation Therapy for ALS.
Meijboom KE, Brown RH. Meijboom KE, et al. Neurotherapeutics. 2022 Jul;19(4):1159-1179. doi: 10.1007/s13311-022-01285-w. Epub 2022 Sep 6. Neurotherapeutics. 2022. PMID: 36068427 Free PMC article. Review.
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
Tran H, Moazami MP, Yang H, McKenna-Yasek D, Douthwright CL, Pinto C, Metterville J, Shin M, Sanil N, Dooley C, Puri A, Weiss A, Wightman N, Gray-Edwards H, Marosfoi M, King RM, Kenderdine T, Fabris D, Bowser R, Watts JK, Brown RH Jr. Tran H, et al. Nat Med. 2022 Jan;28(1):117-124. doi: 10.1038/s41591-021-01557-6. Epub 2021 Dec 23. Nat Med. 2022. PMID: 34949835 Free PMC article.
CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro.
Meijboom KE, Abdallah A, Fordham NP, Nagase H, Rodriguez T, Kraus C, Gendron TF, Krishnan G, Esanov R, Andrade NS, Rybin MJ, Ramic M, Stephens ZD, Edraki A, Blackwood MT, Kahriman A, Henninger N, Kocher JA, Benatar M, Brodsky MH, Petrucelli L, Gao FB, Sontheimer EJ, Brown RH, Zeier Z, Mueller C. Meijboom KE, et al. Nat Commun. 2022 Oct 21;13(1):6286. doi: 10.1038/s41467-022-33332-7. Nat Commun. 2022. PMID: 36271076 Free PMC article.
RNA therapeutics on the rise.
Wang F, Zuroske T, Watts JK. Wang F, et al. Nat Rev Drug Discov. 2020 Jul;19(7):441-442. doi: 10.1038/d41573-020-00078-0. Nat Rev Drug Discov. 2020. PMID: 32341501 No abstract available.
Excessive release of inorganic polyphosphate by ALS/FTD astrocytes causes non-cell-autonomous toxicity to motoneurons.
Arredondo C, Cefaliello C, Dyrda A, Jury N, Martinez P, Díaz I, Amaro A, Tran H, Morales D, Pertusa M, Stoica L, Fritz E, Corvalán D, Abarzúa S, Méndez-Ruette M, Fernández P, Rojas F, Kumar MS, Aguilar R, Almeida S, Weiss A, Bustos FJ, González-Nilo F, Otero C, Tevy MF, Bosco DA, Sáez JC, Kähne T, Gao FB, Berry JD, Nicholson K, Sena-Esteves M, Madrid R, Varela D, Montecino M, Brown RH, van Zundert B. Arredondo C, et al. Neuron. 2022 May 18;110(10):1656-1670.e12. doi: 10.1016/j.neuron.2022.02.010. Epub 2022 Mar 10. Neuron. 2022. PMID: 35276083 Free PMC article.
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
Mueller C, Berry JD, McKenna-Yasek DM, Gernoux G, Owegi MA, Pothier LM, Douthwright CL, Gelevski D, Luppino SD, Blackwood M, Wightman NS, Oakley DH, Frosch MP, Flotte TR, Cudkowicz ME, Brown RH Jr. Mueller C, et al. N Engl J Med. 2020 Jul 9;383(2):151-158. doi: 10.1056/NEJMoa2005056. N Engl J Med. 2020. PMID: 32640133
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