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Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency.
Fernandes-Pires G, Azevedo MD, Lanzillo M, Roux-Petronelli C, Binz PA, Cudalbu C, Sandi C, Tenenbaum L, Braissant O. Fernandes-Pires G, et al. Among authors: tenenbaum l. Mol Ther Methods Clin Dev. 2024 Apr 23;32(2):101251. doi: 10.1016/j.omtm.2024.101251. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38745894 Free PMC article.
83 results