期刊
CURRENT OPINION IN PHARMACOLOGY
卷 34, 期 -, 页码 119-124出版社
ELSEVIER SCI LTD
DOI: 10.1016/j.coph.2017.10.006
关键词
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资金
- Cystic Fibrosis Trust Strategic Research Centre grant [2016-SRC006]
- Cystic Fibrosis Foundation project grant [HART15XXO]
- National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital
- Cystic Fibrosis Trust [VIA049, SRC006] Funding Source: researchfish
- Great Ormond Street Hospital Childrens Charity [V1298] Funding Source: researchfish
Gene therapy for cystic fibrosis (CF) has been the subject of intense research over the last twenty-five years or more, using both viral and liposomal delivery methods, but so far without the emergence of a clinical therapy. New approaches to CF gene therapy involving recent improvements to vector systems, both viral and non-viral, as well as new nucleic acid technologies have led to renewed interest in the field. The field of therapeutic gene editing is rapidly developing with the emergence of CRISPR/Cas9 as well as chemically modified mRNA therapeutics. These new types of nucleic acid therapies are also a good fit with delivery by non -viral delivery approaches which has led to a renewed interest in lipid-based and other nanoformulations.
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