簡単な紹介:
- 著者: Liyan Zhang, Yicheng Lu, Xianmin Wu, Jinyi Fan, Xinru Zhang, Yang Xu, Hao Wei, Qiuhan Sun, Lulu Jiang, Huawei Tuo, Nianci Li, Hua Jin, Wu Cao, Beibei Ye, Ziyu Zhang, Yunkai Zhang, Fangyi Chen, Ling Lu, Jieyu Qi, Renjie Chai, Fangzhi Tan
- ジャーナル: Cell Reports Medicine
- Doi: https://www.doi.org/10.1016/j.xcrm.2026.102725
- 発行日: 2026/3/31
Abstract
Approximately 50% of hereditary deafness cases and most age-related hearing loss result from cochlear hair cell dysfunction. Adeno-associated virus (AAV)-mediated gene therapy offers a promising strategy for hearing restoration. However, the cochlea’s complex cellular composition poses a significant challenge for targeted gene delivery into hair cells. Here, we establish a cross-species screening platform for cochlear AAV tools, applicable from rodents to large animals. Using this platform, we identify human promoters that specifically target inner hair cells, outer hair cells, or all hair cells in mice. The hair cell-specific coProB2 promoter is further validated in Bama miniature pigs and cynomolgus monkeys, achieving specific transgene expression in hair cells without auditory toxicity. Importantly, coProB2-driven gene therapies in DFNB79 and DFNB9 mouse models restore auditory function to near wild-type levels. Thus, this study establishes a cross-species-compatible, hair cell-specific AAV system, providing a versatile toolbox for cochlear hair cell gene therapy.
PackGeneについて
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
