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2024 | 1 |
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Synthetic dosage-compensating miRNA circuits allow precision gene therapy for Rett syndrome.
bioRxiv [Preprint]. 2024 Mar 14:2024.03.13.584179. doi: 10.1101/2024.03.13.584179.
bioRxiv. 2024.
PMID: 38559034
Free PMC article.
Preprint.
Primate-conserved carbonic anhydrase IV and murine-restricted LY6C1 enable blood-brain barrier crossing by engineered viral vectors.
Shay TF, Sullivan EE, Ding X, Chen X, Ravindra Kumar S, Goertsen D, Brown D, Crosby A, Vielmetter J, Borsos M, Wolfe DA, Lam AW, Gradinaru V.
Shay TF, et al.
Sci Adv. 2023 Apr 21;9(16):eadg6618. doi: 10.1126/sciadv.adg6618. Epub 2023 Apr 19.
Sci Adv. 2023.
PMID: 37075114
Free PMC article.
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