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Rescue of glutaric aciduria type I in mice by liver-directed therapies.
Barzi M, Johnson CG, Chen T, Rodriguiz RM, Hemmingsen M, Gonzalez TJ, Rosales A, Beasley J, Peck CK, Ma Y, Stiles AR, Wood TC, Maeso-Diaz R, Diehl AM, Young SP, Everitt JI, Wetsel WC, Lagor WR, Bissig-Choisat B, Asokan A, El-Gharbawy A, Bissig KD. Barzi M, et al. Among authors: gonzalez tj. Sci Transl Med. 2023 Apr 19;15(692):eadf4086. doi: 10.1126/scitranslmed.adf4086. Epub 2023 Apr 19. Sci Transl Med. 2023. PMID: 37075130 Free PMC article.
A humanized mouse model for adeno-associated viral gene therapy.
Barzi M, Chen T, Gonzalez TJ, Pankowicz FP, Oh SH, Streff HL, Rosales A, Ma Y, Collias S, Woodfield SE, Diehl AM, Vasudevan SA, Galvan TN, Goss J, Gersbach CA, Bissig-Choisat B, Asokan A, Bissig KD. Barzi M, et al. Among authors: gonzalez tj. Nat Commun. 2024 Mar 4;15(1):1955. doi: 10.1038/s41467-024-46017-0. Nat Commun. 2024. PMID: 38438373 Free PMC article.
Evaluation of Aav Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy.
Yadav A, Liang R, Press K, Schmidt A, Shabani Z, Leng K, Wang C, Sekhar A, Shi J, Devlin GW, Gonzalez TJ, Asokan A, Su H. Yadav A, et al. Among authors: gonzalez tj. Res Sq [Preprint]. 2024 Jun 12:rs.3.rs-4469011. doi: 10.21203/rs.3.rs-4469011/v1. Res Sq. 2024. Update in: Transl Stroke Res. 2024 Jul 8. doi: 10.1007/s12975-024-01275-4. PMID: 38947073 Free PMC article. Updated. Preprint.
16 results