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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. Among authors: brodsky mh. Nat Commun. 2022 Oct 21;13(1):6286. doi: 10.1038/s41467-022-33332-7. Nat Commun. 2022. PMID: 36271076 Free PMC article.
Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema.
Borel F, Sun H, Zieger M, Cox A, Cardozo B, Li W, Oliveira G, Davis A, Gruntman A, Flotte TR, Brodsky MH, Hoffman AM, Elmallah MK, Mueller C. Borel F, et al. Among authors: brodsky mh. Proc Natl Acad Sci U S A. 2018 Mar 13;115(11):2788-2793. doi: 10.1073/pnas.1713689115. Epub 2018 Feb 16. Proc Natl Acad Sci U S A. 2018. PMID: 29453277 Free PMC article.
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities.
Oikemus SR, Pfister EL, Sapp E, Chase KO, Kennington LA, Hudgens E, Miller R, Zhu LJ, Chaudhary A, Mick EO, Sena-Esteves M, Wolfe SA, DiFiglia M, Aronin N, Brodsky MH. Oikemus SR, et al. Among authors: brodsky mh. Hum Gene Ther. 2022 Jan;33(1-2):25-36. doi: 10.1089/hum.2020.323. Hum Gene Ther. 2022. PMID: 34376056 Free PMC article.
79 results