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Rational engineering of a functional CpG-free ITR for AAV gene therapy.
Pan X, Yue Y, Boftsi M, Wasala LP, Tran NT, Zhang K, Pintel DJ, Tai PWL, Duan D. Pan X, et al. Among authors: wasala lp. Gene Ther. 2022 Jun;29(6):333-345. doi: 10.1038/s41434-021-00296-0. Epub 2021 Oct 6. Gene Ther. 2022. PMID: 34611321 Free PMC article.
AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
Hakim CH, Wasala NB, Nelson CE, Wasala LP, Yue Y, Louderman JA, Lessa TB, Dai A, Zhang K, Jenkins GJ, Nance ME, Pan X, Kodippili K, Yang NN, Chen SJ, Gersbach CA, Duan D. Hakim CH, et al. Among authors: wasala lp. JCI Insight. 2018 Dec 6;3(23):e124297. doi: 10.1172/jci.insight.124297. JCI Insight. 2018. PMID: 30518686 Free PMC article.
Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease.
Hakim CH, Clément N, Wasala LP, Yang HT, Yue Y, Zhang K, Kodippili K, Adamson-Small L, Pan X, Schneider JS, Yang NN, Chamberlain JS, Byrne BJ, Duan D. Hakim CH, et al. Among authors: wasala lp. Mol Ther Methods Clin Dev. 2020 Jul 9;18:664-678. doi: 10.1016/j.omtm.2020.07.004. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775499 Free PMC article.