Acoustically targeted noninvasive gene therapy in large brain volumes
Author:
Funder
John S. Dunn Foundation
Welch Foundation
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.nature.com/articles/s41434-023-00421-1.pdf
Reference71 articles.
1. Turner TJ, Zourray C, Schorge S, Lignani G. Recent advances in gene therapy for neurodevelopmental disorders with epilepsy. J Neurochem. 2021;157:229–62.
2. Simonato M, Bennett J, Boulis NM, Castro MG, Fink DJ, Goins WF, et al. Progress in gene therapy for neurological disorders. Nat Rev Neurol. 2013;9:277–91.
3. Joshi CR, Labhasetwar V, Ghorpade A. Destination brain: the past, present, and future of therapeutic gene delivery. J Neuroimmune Pharmacol. 2017;12:51–83.
4. Keeler GD, Kumar S, Palaschak B, Silverberg EL, Markusic DM, Jones NT, et al. Gene therapy-induced antigen-specific Tregs inhibit neuro-inflammation and reverse disease in a mouse model of multiple sclerosis. Mol Ther. 2018;26:173–83.
5. Spronck EA, Brouwers CC, Vallès A, de Haan M, Petry H, van Deventer SJ, et al. AAV5-miHTT gene therapy demonstrates sustained huntingtin lowering and functional improvement in Huntington disease mouse models. Mol Ther-Methods Clin Dev. 2019;13:334–43.
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