Dendrimers as Non-Viral Vectors in Gene-Directed Enzyme Prodrug Therapy

Molecules

Anca Butuca¹  Maria Totan¹  Felicia Gabriela Gligor¹  Anca Maria Arseniu¹  Claudiu Morgovan¹  Luca-Liviu Rus¹  Anca Maria Juncan¹  Carmen Maximiliana Dobrea¹  Adriana Aurelia Chis¹  Adina Frum¹ 

[1] Preclinical Department, Faculty of Medicine, “Lucian Blaga” University of Sibiu, 550169 Sibiu, Romania;

关键词: GDEPT;    GDEP therapy;    non-viral vector;    dendrimer;    delivery vehicles;    gene delivery system;   

DOI  :  10.3390/molecules26195976

来源: DOAJ

【 摘 要 】

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.

【 授权许可】

Unknown