Challenges in downstream purification of gene therapy viral vectors
Challenges in downstream purification of gene therapy viral vectors
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DOI：
10.1016/j.coche.2021.100780
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发表时间：
2022-03-01
影响因子：
6.6
通讯作者：
Heldt, Caryn L.
中科院分区：
文献类型：
--
作者：
Singh, Nripen;Heldt, Caryn L.
病毒产品的多样性和应用持续爆炸式增长。这些方式可以是疫苗、癌症治疗和基因治疗。然而，它们的多样性也是下游加工的挑战。病毒颗粒可能非常不稳定，因此需要深入的生物学知识来创造稳定的环境。然而，尽管存在这些挑战，我们已经创造了许多产生大量病毒产品的过程。在整个过程中使用不同的净化方法。色谱的新模式正在克服扩散限制珠的许多挑战。基因治疗载体特别关注的是需要将空衣壳与包含感兴趣的治疗基因的完整衣壳分开。随着新的治疗方式的发现，可以通过寻找治疗方法彻底改变护理，病毒治疗的下游加工需要找到解决方案，使这些治疗和治疗负担得起。
The diversity and application of viral products has continued to explode. These modalities can be vaccines, cancer therapies, and gene therapies. However, their diversity is also the challenge in the downstream processing. Viral particles can be very labile, thus requiring intimate knowledge of biology to create environments where they are stable. However, despite these challenges, we have created many processes that produce large amounts of viral products. Different purification methods are utilized throughout the process. New modalities of chromatography are overcoming many of the challenges of diffusion-limited beads. Of special concern for gene therapy vectors is the need to separate the empty capsids from the full capsids, which contain the therapeutic gene of interest. With the discovery of novel therapuetic modalities that could revolutionize care by finding cures, the downstream processing of viral therapies needs to find solutions to make these therapies and cures affordable.