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AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice.
Doyle BM, Turner SMF, Sunshine MD, Doerfler PA, Poirier AE, Vaught LA, Jorgensen ML, Falk DJ, Byrne BJ, Fuller DD. Doyle BM, et al. Among authors: doerfler pa. Mol Ther Methods Clin Dev. 2019 Aug 31;15:194-203. doi: 10.1016/j.omtm.2019.08.009. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31660421 Free PMC article.
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.
Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ. Métais JY, et al. Among authors: doerfler pa. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing.
Leibowitz ML, Papathanasiou S, Doerfler PA, Blaine LJ, Sun L, Yao Y, Zhang CZ, Weiss MJ, Pellman D. Leibowitz ML, et al. Among authors: doerfler pa. Nat Genet. 2021 Jun;53(6):895-905. doi: 10.1038/s41588-021-00838-7. Epub 2021 Apr 12. Nat Genet. 2021. PMID: 33846636 Free PMC article.
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