Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates.
Chen N, Ehmann DE, Crooker R, Derakhchan K, Fang X, Felice B, Galbreath EJ, Glaus C, Gu H, Huang Y, Li C, Li X, Liu N, Palmieri K, Simic D, Sypek J, Thompson S, Winkelmann CT, Choi VW.
Chen N, et al. Among authors: galbreath ej.
Mol Ther Methods Clin Dev. 2023 Mar 28;29:286-302. doi: 10.1016/j.omtm.2023.03.014. eCollection 2023 Jun 8.
Mol Ther Methods Clin Dev. 2023.
PMID: 37359415
Free PMC article.