Mutations in the photoreceptor-specific flippase ABCA4 are associated with Stargardt disease and many other forms of retinal degeneration that currently lack curative therapies. Gene replacement is a logical strategy for ABCA4-associated disease, particularly given the current success of traditional viral-mediated gene delivery, such as with adeno-associated viral (AAV) vectors. However, the la...

Non-viral gene delivery is plagued by low transfection levels compared to viral delivery. The nuclear envelope presents a significant obstacle for non-viral vectors. A peptide-based nuclear localizing sequence has been incorporated into non-viral vectors to traverse the nuclear envelope. Here, we selected a photo-chemical method for covalently labeling the peptide onto plasmid DNA. The hypothes...

The goal of tissue engineering is the production of functional, biocompatible tissues by seeding cells within biological or synthetic scaffolds. One tissue engineering approach involves the genetic modification of cells that are seeded onto (or into) scaffolds prior to implantation. The genetic modification is achieved through gene delivery, with can utilize viral transduction or non-viral tran...

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...

BACKGROUND The cell source for transplantation therapy is always a prerequisite question to be solved in clinical applications. Neural cells are considered non-regenerable, which highly restrict their application in the treatment for nerve injury. Therefore, neural trans-differentiation based on gene transfection provides a new solution to this issue. Compared to viral strategy, non-viral gene ...

Gene delivery of nucleic acid to the cell nucleus is a fundamental step in gene therapy. In this review of modeling drug and gene delivery, we focus on the particular stage of plasmid DNA or virus cytoplasmic trafficking. A challenging problem is to quantify the success of this limiting stage. We present some models and simulations of plasmid trafficking and of the limiting phase of DNA-polycat...

Objective: Transient expression of therapeutic genes within lung allografts may modulate the pathological processes following allotransplantation. Whilst ef®cient gene transfer to lungs has been reported with viral vectors, their usefulness is limited on the grounds of safety. Since non-viral systems overcome many of these safety issues, our studies were designed to evaluate the ef®ciency of se...

Microbubbles and ultrasound have recently been investigated with a view to improving the transfection efficiency of non-viral gene delivery systems. However, microbubbles are unstable and their targeting ability is insufficient for clinical use. To circumvent these problems, we developed novel polyethyleneglycol (PEG) modified liposomes (Bubble liposomes) containing perfluoropropane, which is a...

chitosan-graft-polyethylenimine (chi-g-pei) copolymer has been used for theimprovement of low transfection efficiency of chitosan. the present study aims to test thepulmonary toxicity and efficiency of chi-g-pei as an aerosol gene carrier. mice were exposedto aerosol containing green-fluorescent protein (gfp)-polyethylenimine (pei) or gfp-chig-pei complexes for 30 min during the development of ...

Theoretically, cystic fibrosis transmembrane conductance regulator (CFTR) gene replacement during the neonatal period can decrease morbidity and mortality from cystic fibrosis (CF). In vivo gene transfers have been accomplished in CF patients. Choice of vector, mode of delivery to airways, translocation of genetic information, and sufficient expression level of the normalized CFTR gene are issu...