The AAPS Journal

, 11:671

Nonviral Gene Delivery: Principle, Limitations, and Recent Progress

Review Article Theme: Emerging Drug Delivery Technologies

DOI: 10.1208/s12248-009-9143-y

Cite this article as:
Al-Dosari, M.S. & Gao, X. AAPS J (2009) 11: 671. doi:10.1208/s12248-009-9143-y

Abstract

Gene therapy is becoming a promising therapeutic modality for the treatment of genetic and acquired disorders. Nonviral approaches as alternative gene transfer vehicles to the popular viral vectors have received significant attention because of their favorable properties, including lack of immunogenicity, low toxicity, and potential for tissue specificity. Such approaches have been tested in preclinical studies and human clinical trials over the last decade. Although therapeutic benefit has been demonstrated in animal models, gene delivery efficiency of the nonviral approaches remains to be a key obstacle for clinical applications. This review focuses on existing and emerging concepts of chemical and physical methods for delivery of therapeutic nucleic acid molecules in vivo. The emphasis is placed on discussion about problems associated with current nonviral methods and recent efforts toward refinement of nonviral approaches.

Key words

gene deliverygene therapylipoplexnonviral vectorspolyplextransfection

Copyright information

© American Association of Pharmaceutical Scientists 2009

Authors and Affiliations

  1. 1.Department of Pharmacognosy, College of PharmacyKing Saud UniversityRiyadhSaudi Arabia
  2. 2.Department of Pharmaceutical Sciences, School of PharmacyUniversity of PittsburghPittsburghUSA