Cationic Polyelectrolyte Vectors in Gene Delivery
Gene therapy holds great promise and potential for treating diseases arising from genetic disorders whether it is acquired or inherited. Cationic polymers are widely used for gene delivery applications as they can form nanoplexes readily with DNA, an anio
- PDF / 248,023 Bytes
- 23 Pages / 439.37 x 666.142 pts Page_size
- 59 Downloads / 187 Views
Abstract
Gene therapy holds great promise and potential for treating diseases arising from genetic disorders whether it is acquired or inherited. Cationic polymers are widely used for gene delivery applications as they can form nanoplexes readily with DNA, an anionic polyelectrolyte that can be delivered intracellularly with ease. Till date a variety of numerous cationic polymers of varying molecular weight, structure, functional groups, composition and charge density are developed for gene delivery applications. These polymers can be broadly classified into synthetic, natural and graft polymers. The major barriers associated with non-viral vectors include its toxicity, evading immune system, hemocompatibility, efficient intracellular delivery at target site, intracellular unpacking and ultimately the gene expression. The first report on the usage of cationic polymer as gene delivery vector dates back to three decades. Though cationic polymers are now well established as a successful gene delivery vector, those reaching to clinical trials are still limited owing to above mentioned barriers. The chapters of this book will focus on the most successful cationic polymeric systems of both natural and synthetic origin, design concerns and also the recent advances in the polymer design approach for a successful gene transfer. Keywords
Non-viral gene delivery Cationic polymers
Redox sensitive polymers
Graft polysaccharides
M.C.D. Sherly S.S. Priya M.R. Rekha (&) Division of Biosurface Technology, Biomedical Technology Wing, Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram 695012, Kerala, India e-mail: [email protected] © Springer Nature Singapore Pte Ltd. 2017 S. Jana and S. Jana (eds.), Particulate Technology for Delivery of Therapeutics, DOI 10.1007/978-981-10-3647-7_12
395
396
1
M.C.D. Sherly et al.
Introduction
Millions of people are affected by various genetic disorders be it a hereditary or acquired one. Gene therapy is a promising approach to replace the faulty genes with or introduce a functional one so as to cure or treat a particular clinical condition. In gene therapy gene is the medicine that needs to be delivered intracellularly. To introduce a foreign gene into a living system is not a simple task and needs careful designing of vectors for a successful therapeutic application. Initially when the concept of gene therapy was introduced viruses were chosen as gene delivery vectors owing to their high transfection ability. Various viral vectors used for clinical trials include adenovirus, adeno-associated virus, retrovirus, lentivirus, herpes simplex virus and so on (Verma and Somia 1997; During 1997; Vile et al. 1996). In spite of their advantage as suitable gene carrier, there is large number of problems associated with both in vivo and in vitro applications of these vectors. This mainly include the induction of immune response by the viral proteins, insertional mutagenesis, recombination with wild type viruses, limitation in the size of DNA to be car
Data Loading...
