The effective and sustained delivery of DNA and siRNAs locally would increase the applicability of gene therapy in tissue regeneration and cancer therapy. One promising approach is to use hydrogel scaffolds to encapsulate and deliver nucleotides in the form of nanoparticles to the disease sites. However, this approach is currently limited by the inability to load concentrated and active gene de...

Gene therapy offers important perspectives in current and future medicine but suffers from imperfect vectors for the delivery of the therapeutic gene. Most preclinical and clinical trials have been based on the use of viral vectors, which have evident advantages but also some serious disadvantages. In the past decade the use of DNA transposon-based systems for gene delivery has emerged as a non...

Non-viral gene therapy offers the potential to deliver nucleic acids producing therapeutic proteins to treat genetic diseases without the limitations observed with viral vectors. Before the therapeutic potential of non-viral gene delivery can be realized, several barriers to efficient gene delivery must be overcome. One delivery barrier of interest is the enhancement of endosomal escape to prev...

objective(s): the introduction of nucleic acids into cells for therapeutic objectives is significantly hindered by the size and charge of these molecules and therefore requires efficient vectors that assist cellular uptake. for several years great efforts have been devoted to the study of development of recombinant vectors based on biological domains with potential applications in gene therapy....

abstract among synthetic carriers, dendrimers with the more flexible structure have attracted a great deal of researchers' attention in the field of gene delivery. followed by the promising results upon hydrophobic modification on polymeric structures in our laboratory, alkylcarboxylated poly (propylenimine)-based carriers were synthesized by nucleophilic substitution of amines with alkyl moiet...

conclusions eudragit coated nanoparticle of chitosan is an efficient choice for oral delivery of dna to upper part of gi tract. results the mean size and zeta potential of particles were 300 ± 4 nm and 14 ± 0.5 mv, respectively. encapsulation of this system was 89.6 ± 1.2%. dna release from batches was less than 12% at ph = 5.2 and more than 60% at ph = 6.8 with significant difference of p < 0....

DNA based transposon vectors offer a mechanism for non-viral gene delivery into mamma‐ lian and human cells. These vectors work via a cut-and-paste mechanim whereby transpo‐ son DNA containing a transgene(s) of interest is integrated into chromosomal DNA by a transposase enzyme. The first DNA based transposon system which worked efficienty in human cells was sleeping beauty. This was followed a...

Non-viral gene delivery systems are being developed to address limitations of viral gene delivery. Many of these non-viral systems are modeled on the properties of viruses including cell surface binding, endocytosis, endosomal escape, and nuclear targeting. Most non-viral gene transfer systems exhibit little correlation between in vitro and in vivo efficiency, hampering a systematic approach to...

Ocular gene therapy holds promise for the treatment of numerous blinding disorders. Despite the significant progress in the field of viral and non-viral gene delivery to the eye, significant obstacles remain in the way of achieving high-level transgene expression without adverse effects. The retinal pigment epithelium (RPE) is involved in the pathogenesis of retinal diseases and is a key target...