Current Gene Therapy - Volume 11, Issue 1, 2011

This special issue is devoted to artificial nucleases engineered for targeted genome modification. The discovery of restriction enzymes in the late 60s paved the way for enzymatic manipulation of DNA in the test tube. The novel reagents reviewed in this special issue now promise to usher us into a new era of DNA modification performed directly in living cells. Artificial nucleases are designed to introduce a targeted cut in geno Read More

Zinc-finger nucleases (ZFNs) are emerging as very powerful tools for directed genome modifications. Their key features are: a DNA-binding domain comprised of zinc fingers that can be designed to favor very specific targets; a nonspecific cleavage domain that must dimerize to cut DNA - this requirement enhances specificity and minimizes random cleavage. ZFNs have been shown to be effective in a wide range of organisms Read More

The importance of safer approaches for gene therapy has been underscored by a series of severe adverse events (SAEs) observed in patients involved in clinical trials for Severe Combined Immune Deficiency Disease (SCID) and Chromic Granulomatous Disease (CGD). While a new generation of viral vectors is in the process of replacing the classical gamma-retrovirus-based approach, a number of strategies have e Read More

Therapeutic genome engineering is a hallmark of gene therapy but only recent technological advances have permitted the modification of complex genomes in a targeted fashion. Zinc-finger nucleases (ZFNs) have developed into a major playmaker in the genome engineering field and have been employed to trigger the targeted editing of genomes at over 50 gene loci in 11 model organisms, including fruitfly, zebrafish and r Read More

Homologous recombination is almost the only way to modify the genome in a predetermined fashion, despite its quite low frequency in mammalian cells. It has been already reported that the frequency of this biological process can be notably increased by inducing a double strand break (DSB) at target site. This article presents completely chemistrybased artificial restriction DNA cutter (ARCUT) for the promotion of homologous re Read More

Mesenchymal stem cells (MSCs) can be isolated from several tissues in the body, have the ability to selfrenewal, show immune suppressive properties and are multipotent, being able to generate various cell types. At present, due to their intrinsic characteristics, MSCs are considered very promising in the area of tissue engineering and regenerative medicine. In this context, genetic modification can be a powerful tool to co Read More

The success of gene therapy largely relies on the development of high-efficient and low-toxic gene delivery vectors. Nanovector-based delivery of nucleic acids is a very promising approach for the effective transfer of genetic materials into cells. Compared with encapsulating of nucleic acids inside biodegradable nanoparticles which often suffers from low encapsulation efficiency and degradation of the loaded therapeutic gen Read More