Abstract
Adeno-associated virus (AAV) vectors have gained tremendous attention as in vivo delivery systems in gene therapy for inherited monogenetic diseases. First market approvals, excellent safety data, availability of large-scale production protocols, and the possibility to tailor the vector towards optimized and cell-type specific gene transfer offers to move from (ultra) rare to common diseases. Cancer, a major health burden for which novel therapeutic options are urgently needed, represents such a target. We here provide an up-to-date overview of the strategies which are currently developed for the use of AAV vectors in cancer gene therapy and discuss the perspectives for the future translation of these pre-clinical approaches into the clinic.
Highlights
Cancer represents a major health problem worldwide
The liver can be affected by tumor formation either originating from liver-resident cells such as hepatocytes or from tumor cells derived from distant tumors that engraft in the liver to form metastasis
It appears that first generation associated virus (AAV) vectors are especially suitable for targeting liver and central nervous system (CNS) related cancers for tumor cell-directed treatments due to the favorable gene transfer efficacy of certain natural AAV serotypes
Summary
Cancer represents a major health problem worldwide. In 2018, the global incidence was as high as 17,000,000 new cases and more than 9,400,000 patients died from cancer With an increasing understanding of cancer biology, these initial six hallmarks were updated to include four additional ones [2] These cancer hallmarks served as a blueprint for the development of new treatment strategies, an impressive number of which has made their way from preclinical development to the clinic (Table 1). The latest developments in cancer immunotherapy have opened new exciting avenues for the development of cancer gene therapy In addition to their ability to introduce novel genes into target cells, a number of gene therapy vectors are prime candidates for the development of anti-cancer vaccines or oncolytic virotherapy, and some have already reached the clinic [3]. Inhibition of angiogenic pathways (i.e., VEGF, PlGF): monoclonal antibodies, fusion constructs, tyrosine kinase inhibitors. We will focus on the role of adeno-associated virus (AAV) vectors, by giving a comprehensive overview over latest developments and the perspectives we envision for the use of this vector system in the context of the rapidly evolving field of cancer research and treatment
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