期刊
MOLECULES
卷 26, 期 19, 页码 -出版社
MDPI
DOI: 10.3390/molecules26195976
关键词
GDEPT; GDEP therapy; non-viral vector; dendrimer; delivery vehicles; gene delivery system; transgene; targeted therapy
Gene-directed enzyme prodrug therapy (GDEPT) is a promising strategy for prodrug delivery, with benefits including enhanced efficacy and reduced off-target toxicity. Non-viral vectors, such as dendrimers, have gained interest due to their decreased immunogenicity and high specificity for delivering genes in GDEPT therapy.
Gene-directed enzyme prodrug therapy (GDEPT) has been intensively studied as a promising new strategy of prodrug delivery, with its main advantages being represented by an enhanced efficacy and a reduced off-target toxicity of the active drug. In recent years, numerous therapeutic systems based on GDEPT strategy have entered clinical trials. In order to deliver the desired gene at a specific site of action, this therapeutic approach uses vectors divided in two major categories, viral vectors and non-viral vectors, with the latter being represented by chemical delivery agents. There is considerable interest in the development of non-viral vectors due to their decreased immunogenicity, higher specificity, ease of synthesis and greater flexibility for subsequent modulations. Dendrimers used as delivery vehicles offer many advantages, such as: nanoscale size, precise molecular weight, increased solubility, high load capacity, high bioavailability and low immunogenicity. The aim of the present work was to provide a comprehensive overview of the recent advances regarding the use of dendrimers as non-viral carriers in the GDEPT therapy.
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