簡(jiǎn)介:
- 作者: Haoyu Wu, Ye Bu, Lang He, Dong Yao, Huapeng Li & Wenguang Yin
- 雜志: Virology Journal
- 出版日期: 2024 Nov 25
摘要
Background
Gene therapy holds great potential for treating various acquired and inherited pulmonary diseases. Adeno-associated viral (AAV) vectors have been thought to be primary candidates for gene delivery in patients with pulmonary diseases. However, the tropism of AAVs in the lungs remains largely unknown.
Results
Here, we investigate the tropism of twenty serotypes of AAVs by examining AAV-packed vector expression of the enhanced green fluorescent protein (eGFP) in mice. AAV1, AAV4, AAV5, AAV6, AAV6.2, AAV-PHP.B, and AAV-PHP.S exhibit high transduction rates in the airway epithelium. AAV1, AAV4, AAV5, AAV6, and AAV6.2 highly infect club cells. AAV1, AAV4, AAV5, AAV6, AAV6.2, and AAV-PHP.B efficiently infect ciliated cells. AAV8 and AAVrh10 can infect a few alveolar type I cells. AAV1, AAV5, AAV6, AAV6.2, AAV9, and AAVie can infect alveolar type II cells. AAV1, AAV5, AAVie, AAV-PHP.B, AAV-PHP.eB, and AAV-PHP.S can infect a few endothelial cells. However, none of these AAVs can efficiently infect neuroendocrine or smooth muscle cells.
Conclusions
Our findings provide comprehensive information about the tropism of AAVs in pulmonary epithelium in mice, which might be helpful in developing efficient AAV-mediated gene therapy strategies for pulmonary disease treatment.
關(guān)于派真
作為一家專(zhuān)注于AAV 技術(shù)十余年，深耕基因治療領(lǐng)域的CRO&CDMO，派真生物可提供從載體設(shè)計(jì)、構(gòu)建到 AAV、慢病毒和 mRNA 服務(wù)的一站式解決方案。憑借深厚的技術(shù)實(shí)力、卓越的運(yùn)營(yíng)管理和高標(biāo)準(zhǔn)的服務(wù)交付，我們?yōu)槿蚩蛻?hù)提供一站式CMC解決方案，包括從早期概念驗(yàn)證、成藥性評(píng)估到IIT、IND及BLA的各個(gè)階段。
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憑借我們獨(dú)立知識(shí)產(chǎn)權(quán)的π-alphaTM 293 細(xì)胞AAV高產(chǎn)技術(shù)平臺(tái)，我們能將AAV產(chǎn)量提高多至10倍，每批次產(chǎn)量可達(dá)1×101?vg，以滿(mǎn)足多樣化的商業(yè)化和臨床項(xiàng)目需求。此外，我們定制化的mRNA和脂質(zhì)納米顆粒（LNP）產(chǎn)品及服務(wù)覆蓋藥物和疫苗開(kāi)發(fā)的各個(gè)階段，從研發(fā)到符合GMP的生產(chǎn)，提供端到端的一站式解決方案。
