CRISPR-Cas9 Engineered Extracellular Vesicles for the Treatment of Dominant Progressive Hearing Loss
X. Pan, P. Huang, S. Ali, B. Renslo, T. E. Hutchinson, N. Erwin, Z. F. Greenberg, Z. Ding, Y. Li, A. Warnecke, N. E. Fernandez, H. Staecker, M. He
The paper develops a scalable microfluidic electroporation system to load gRNA:Cas9 RNP complexes into extracellular vesicles, achieving effective gene therapy for hearing loss in a mouse model.
bioRxiv · Sep 14, 2023 · PreprintBioengineering