The advances and challenges of Gene Therapy for Duchenne Muscular Dystrophy

The last ten years have been characterized by unprecedented breakthroughs in the field of biology. This extraordinary development is the result of several achievements, one of the most significant of which was the culmination of the Human Genome Project (PGH) in 2004. This project, which lasted more than ten years and which mobilized considerable human and financial resources for sequencing the entire human genome. This gigantic project was made possible thanks to the progress of sequencing technology and bioinformatics [1].Read more....

Progress in the development of Lipoplex and Polyplex modified with Anionic Polymer for efficient Gene Delivery

INTRODUCTION

Nucleic acid-based therapy (gene therapy) has recently received much attention as a therapeutic methodology for a number of acquired and inherited diseases [1]. Nucleic acid-based therapy can be defined as the treatment of a disease by the transfer of a therapeutic gene (DNA or RNA) into target tissues for expression or suppression of a gene related to the disease process of interest.Read more....

CRISPR genome editing: A general view

CRISPR technology has presented a path forward for genomic engineering and gene modification. The framework for the use of CRISPR technology to manipulate the human genome is of great interest and the form of its development and application has excited the researchers and biotech communities as the number of publications citing CRISPR gene targeting system has rose predominantly as indexed in PubMed. From a technical standpoint of view, most of us think that this would be relatively straightforward process, but technical feasibility is never the only consideration in doing experiments. Much of the discussion about CRISPR engineering has revolved mostly around its ability for treating disease or editing the genes of human embryos. In the real sense, what the biologists desire about CRISPR is its specificity: the ability to target and determine particular DNA sequences in the genome circuit.Read more....