Gene Therapy Progress and Prospects – Vectorology: design and production of expression cassettes in AAV vectors
Date: 2.3.2006
Adeno-associated virus (AAV) derived vectors are considered highly eligible vehicles for human gene therapy.
Not only do they possess many great potential for clinical applications due to their wide range of tissue targets but also their excellent preclinical safety profile makes them particularly suitable candidates for treating serious diseases. Initial clinical trials have yielded encouraging results and prompted further improvements in their design and methods of production.
Many studies have been performed to modify the tropism of recombinant (r)AAV by capsid modification. However, the precise control of spatial and temporal gene expression, which may be important in determining the safety and efficacy of gene transfer, lies in a rational choice and a subtle combination of various regulatory genetic elements to be inserted into the expression cassette.
Moreover, new strategies based on such genetic sequences open new perspectives for enhancing vector genome persistence, disrupting or reducing pathogenic gene expression and even targeting genes.
"Source":[ http://www.nature.com/gt/journal/vaop/ncurrent/abs/3302724a.html].
Related articles
Gene therapy for foetuses hope - The work is controversial not just because of the ethics but also safety concerns (20.3.2007)
DNA nanoparticles hold promise in gene therapy for Parkinson's disease - University of Kentucky researcher David Yurek was recently awarded $66,000 by The Michael J (3.3.2007)
Internalization of novel non-viral vector TAT-streptavidin into human cells - Background The cell-penetrating peptide derived from the Human immunodeficiency virus-1 transactivator protein Tat possesses the capacity to promote the effective uptake of various cargo molecules across the plasma membrane in vitro and in vivo (12.2.2007)
