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Genome Editing for Mucopolysaccharidoses.
Poletto E, Baldo G, Gomez-Ospina N. Poletto E, et al. Int J Mol Sci. 2020 Jan 13;21(2):500. doi: 10.3390/ijms21020500. Int J Mol Sci. 2020. PMID: 31941077 Free PMC article. Review.
CNS Repopulation by Hematopoietic-Derived Microglia-Like Cells Corrects Progranulin deficiency.
Colella P, Sayana R, Suarez-Nieto MV, Sarno J, Nyame K, Xiong J, Vera LNP, Basurto JA, Corbo M, Limaye A, Davis KL, Abu-Remaileh M, Gomez-Ospina N. Colella P, et al. Res Sq [Preprint]. 2023 Sep 12:rs.3.rs-3263412. doi: 10.21203/rs.3.rs-3263412/v1. Res Sq. 2023. Update in: Nat Commun. 2024 Jul 5;15(1):5654. doi: 10.1038/s41467-024-49908-4 PMID: 37790525 Free PMC article. Updated. Preprint.