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CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder.
Calias P, Papisov M, Pan J, Savioli N, Belov V, Huang Y, Lotterhand J, Alessandrini M, Liu N, Fischman AJ, Powell JL, Heartlein MW. Calias P, et al. Among authors: lotterhand j. PLoS One. 2012;7(1):e30341. doi: 10.1371/journal.pone.0030341. Epub 2012 Jan 18. PLoS One. 2012. PMID: 22279584 Free PMC article.
Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH.
Ahmed SS, Rubin H, Wang M, Faulkner D, Sengooba A, Dollive SN, Avila N, Ellsworth JL, Lamppu D, Lobikin M, Lotterhand J, Adamson-Small L, Wright T, Seymour A, Francone OL. Ahmed SS, et al. Among authors: lotterhand j. Mol Ther Methods Clin Dev. 2020 Mar 13;17:568-580. doi: 10.1016/j.omtm.2020.03.009. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32258219 Free PMC article.
Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS.
Smith LJ, Schulman LA, Smith S, Van Lieshout L, Barnes CM, Behmoiras L, Scarpitti M, Kivaa M, Duong KL, Benard LO, Ellsworth JL, Avila N, Faulkner D, Hayes A, Lotterhand J, Rivas JI, Sengooba AV, Tzianabos A, Seymour AB, Francone OL. Smith LJ, et al. Among authors: lotterhand j. Mol Ther Methods Clin Dev. 2022 Jun 30;26:224-238. doi: 10.1016/j.omtm.2022.06.013. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 35859693 Free PMC article.
A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes.
van Lieshout LP, Rubin M, Costa-Grant K, Ota S, Golebiowski D, Panico T, Wiberg E, Szymczak K, Gilmore R, Stanvick M, Burnham B, Gagnon J, Iwuchukwu I, Yang G, Ghazi I, Meola A, Dickerson R, Thiers T, Mustich L, Hayes A, Rivas I, Lotterhand J, Avila N, McGivney J, Yin J, Kelly T. van Lieshout LP, et al. Among authors: lotterhand j. Mol Ther Methods Clin Dev. 2023 May 8;29:426-436. doi: 10.1016/j.omtm.2023.05.004. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273900 Free PMC article.
Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice.
St Martin T, Seabrook TA, Gall K, Newman J, Avila N, Hayes A, Kivaa M, Lotterhand J, Mercaldi M, Patel K, Rivas IJ, Woodcock S, Wright TL, Seymour AB, Francone OL, Gingras J. St Martin T, et al. Among authors: lotterhand j. J Neurosci. 2023 May 10;43(19):3567-3581. doi: 10.1523/JNEUROSCI.1829-22.2023. Epub 2023 Mar 28. J Neurosci. 2023. PMID: 36977578 Free PMC article.