期刊
TRENDS IN BIOTECHNOLOGY
卷 39, 期 5, 页码 474-487出版社
CELL PRESS
DOI: 10.1016/j.tibtech.2020.07.012
关键词
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资金
- Canadian Institute of Health Research [CIHR PJT166035]
- Faculty of Pharmaceutical Sciences of the University of British Columbia
- NanoMedicines Innovation Network (NMIN)
- Sao Paulo Research Foundation (FAPESP) [2017/244021, 2019/051000]
- Swiss National Science Foundation [183923]
- Lohn Foundation
Gene therapies are powerful in treating diseases, but face challenges in skin disorders due to the unique barrier properties of human skin that limit the efficient delivery of nucleic acid payloads. Delivery strategies are identified as the major obstacle in fully utilizing the potential of gene therapies for various human diseases, not just skin disorders.
Gene therapies are powerful tools to prevent, treat, and cure human diseases. The application of gene therapies for skin diseases received little attention so far, despite the easy accessibility of skin and the urgent medical need. A major obstacle is the unique barrier properties of human skin, which significantly limits the absorption of biomacromolecules, and thus hampers the efficient delivery of nucleic acid payloads. In this review, we discuss current approaches, successes, and failures of cutaneous gene therapy and provide guidance toward the development of next-generation concepts. We specifically allude to the delivery strategies as the major obstacle that prevents the full potential of gene therapies ? not only for skin disorders but also for almost any other human disease.
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