Vector Targeting in Gene Therapy

Abstract

In gene therapy, current vector systems demonstrate insufficient expression and lack of specificity, resulting in poor curative effects in target cells and/or high toxicity to nontarget cells. Thus, vector optimization is critical for the derivation of efficient clinical gene therapeutics. Recently, adenoviral vectors have been targeted at the level of transduction and transcription to overcome these issues. Using genetic capsid modification techniques, adapter molecules with tissue-specific antibodies and tissue-specific promoters (TSPs), these vectors can be efficiently targeted. These strategies are applicable for many types of target cells and are compatible with a variety of targeting molecules. These combinations are aimed at improving therapeutic index of diseases such as cancers, as well as nonmalignant diseases such as pulmonary hypertension.