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A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Tordo J, O'Leary C, Antunes ASLM, Palomar N, Aldrin-Kirk P, Basche M, Bennett A, D'Souza Z, Gleitz H, Godwin A, Holley RJ, Parker H, Liao AY, Rouse P, Youshani AS, Dridi L, Martins C, Levade T, Stacey KB, Davis DM, Dyer A, Clément N, Björklund T, Ali RR, Agbandje-McKenna M, Rahim AA, Pshezhetsky A, Waddington SN, Linden RM, Bigger BW, Henckaerts E. Tordo J, et al. Among authors: henckaerts e. Brain. 2018 Jul 1;141(7):2014-2031. doi: 10.1093/brain/awy126. Brain. 2018. PMID: 29788236 Free PMC article.
AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.
Hughes MP, Smith DA, Morris L, Fletcher C, Colaco A, Huebecker M, Tordo J, Palomar N, Massaro G, Henckaerts E, Waddington SN, Platt FM, Rahim AA. Hughes MP, et al. Among authors: henckaerts e. Hum Mol Genet. 2018 Sep 1;27(17):3079-3098. doi: 10.1093/hmg/ddy212. Hum Mol Genet. 2018. PMID: 29878115 Free PMC article.
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.
Karda R, Rahim AA, Wong AMS, Suff N, Diaz JA, Perocheau DP, Tijani M, Ng J, Baruteau J, Martin NP, Hughes M, Delhove JMKM, Counsell JR, Cooper JD, Henckaerts E, Mckay TR, Buckley SMK, Waddington SN. Karda R, et al. Among authors: henckaerts e. Sci Rep. 2020 Feb 7;10(1):2121. doi: 10.1038/s41598-020-59075-3. Sci Rep. 2020. PMID: 32034258 Free PMC article.
Comparative structural, biophysical, and receptor binding study of true type and wild type AAV2.
Bennett A, Hull J, Jolinon N, Tordo J, Moss K, Binns E, Mietzsch M, Hagemann C, Linden RM, Serio A, Chipman P, Sousa D, Broecker F, Seeberger P, Henckaerts E, McKenna R, Agbandje-McKenna M. Bennett A, et al. Among authors: henckaerts e. J Struct Biol. 2021 Dec;213(4):107795. doi: 10.1016/j.jsb.2021.107795. Epub 2021 Sep 10. J Struct Biol. 2021. PMID: 34509611 Free PMC article.
35 results