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Leveraging gene therapy to achieve long-term continuous or controllable expression of biotherapeutics.
Cripe TP, Hutzen B, Currier MA, Chen CY, Glaspell AM, Sullivan GC, Hurley JM, Deighen MR, Venkataramany AS, Mo X, Stanek JR, Miller AR, Wijeratne S, Magrini V, Mardis ER, Mendell JR, Chandler DS, Wang PY. Cripe TP, et al. Among authors: chandler ds. Sci Adv. 2022 Jul 15;8(28):eabm1890. doi: 10.1126/sciadv.abm1890. Epub 2022 Jul 13. Sci Adv. 2022. PMID: 35857488 Free PMC article.
In addition, we created an inducible version using an exon skipping strategy and achieved repeated, on-demand expression up to at least 36 weeks after AAV injection. Our system could be considered for short-term and/or repeated expression of other transgenes of interest fo …
In addition, we created an inducible version using an exon skipping strategy and achieved repeated, on-demand expression up to at least 36 w …