Recent advances in designing safe and efficient viral vectors have spearheaded unprecedented growth clinical trials regulatory approvals for experimental gene therapies. Still, important challenges remain therapies that require systemic administration into the circulation, including patient’s immune response need precise activation target cell population while sparing other tissues. Extensive r...

The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the parvovirus family. The advantages of this vector system is the stability of the viral capsid, the low immunogenicity, the ability to transduce both div...

Gene transfer within the cardiovascular system was first demonstrated in 1989 yet, despite extensive basic-science and clinical research, unequivocal benefit in the clinical setting remains to be demonstrated. Potential reasons for this include the fact that recombinant viral vectors, used in the majority of clinical studies, have inherent problems with immunogenicity that are difficult to circ...

Over the last few years the development of plant viral vectors for protein expression has made rapid and impressive progress. Plant viruses are versatile vectors for production of proteins since they are easy to manipulate, quick to evaluate and offer the possibility of great production yields. They are particularly powerful expression systems for the production of recombinant proteins and pept...

Elevated intraocular pressure is the primary cause of open angle glaucoma. Outflow resistance exists within the trabecular meshwork but also at the level of Schlemm's canal and further downstream within the outflow system. Viral vectors allow to take advantage of naturally evolved, highly efficient mechanisms of gene transfer, a process that is termed transduction. They can be produced at biosa...

Lentiviral vectors (LV) are efficient vehicles for gene transfer in mammalian cells due to their capacity to stably express a gene of interest in non-dividing and dividing cells. Their use has exponentially grown in the last years both in research and in gene therapy protocols, reaching 12% of the viral vector based clinical trials in 2011 [1]. This chapter reviews and discusses the state of th...

1. Abstract 2. Introduction 3. Viral vectors 3.1. Adenoviral vectors 3.2. Adeno-associatedvectors 3.3. Retroand Lentiviral vectors 3.4. Herpes viral vectors 4. Chemical gene transfer 5. Physical methods for gene delivery 5.1. Ballistic gene delivery 5.2. Micorseeding 5.3. Electroporation 6. Gene regulation 7. Gene silencing and transcriptional factors 8. Clinical trials 9. Perspective 10. Refer...

The rational design of effective and safe non-viral gene vectors is largely dependent on the understanding of the structure-property relationship. We herein report the design of a new series of cationic, α-helical polypeptides with different side charged groups (amine and guanidine) and hydrophobicity, and mechanistically unraveled the effect of polypeptide structure on the gene delivery capabi...

DNA therapy for cancer requires efficient, selective and safe DNA delivery systems. Compared with other non-viral methods such as lipid or polymer-based DNA delivery vectors, peptide-based DNA delivery systems are biocompatible and biodegradable, which leads to lower immunogenicity and lower toxicity. Moreover, peptide vectors are easier to produce and their compositions easier to control becau...

The human immunodeficiency virus (HIV) infects primarily the hematopoietic and immune systems. At the onset of infection, an initial activation of the immune system occurs, with a subsequent suppression thereafter due to direct viral infection of cells, inhibitory effects of HIV proteins, an altered microenvironment with cytokine imbalance, and increased apoptosis of both infected and non-infec...