fulltext.study @t Gmail

Gene therapy, gene targeting and induced pluripotent stem cells: Applications in monogenic disease treatment

Paper ID Volume ID Publish Year Pages File Format Full-Text
14360 1213 2011 10 PDF Available
Title
Gene therapy, gene targeting and induced pluripotent stem cells: Applications in monogenic disease treatment
Abstract

Monogenic diseases are often severe, life-threatening disorders for which lifelong palliative treatment is the only option. Over the last two decades, a number of strategies have been devised with the aim to treat these diseases with a genetic approach. Gene therapy has been under development for many years, yet suffers from the lack of an effective and safe vector for the delivery of genetic material into cells. More recently, gene targeting by homologous recombination has been proposed as a safer treatment, by specifically correcting disease-causing mutations. However, low efficiency is a major drawback. The emergence of two technologies could overcome some of these obstacles. Terminally differentiated somatic cells can be reprogrammed, using defined factors, to become induced pluripotent stem cells (iPSCs), which can undergo efficient gene mutation correction with the aid of fusion proteins known as zinc finger nucleases (ZFNs). The amalgamation of these two technologies has the potential to break through the current bottleneck in gene therapy and gene targeting.

Keywords
Monogenic diseases; Gene therapy; Gene targeting; Induced pluripotent stem cells; Reprogramming; Zinc finger nucleases
First Page Preview
Gene therapy, gene targeting and induced pluripotent stem cells: Applications in monogenic disease treatment
Publisher
Database: Elsevier - ScienceDirect
Journal: Biotechnology Advances - Volume 29, Issue 1, January–February 2011, Pages 1–10
Authors
, ,
Subjects
Physical Sciences and Engineering Chemical Engineering Bioengineering