Benedetti, Sara;
Cossu, Giulio;
Tedesco, Francesco Saverio;
(2015)
Gene and Cell Therapies for Muscular Dystrophies.
In: Templeton, Nancy Smyth, (ed.)
Gene and Cell Therapy: Therapeutic Mechanisms and Strategies.
CRC Press: Boca Raton, Florida, USA.
Preview |
Text
Benedetti_gene and cell.pdf Download (776kB) | Preview |
Abstract
The main goal of gene and/or cell therapy is to repair and/or replace the mutated gene responsible for a genetic disease, thus reverting once and (ideally) forever the pathological phenotype. This is an exceptionally challenging aim to achieve in the case of muscular dystrophies, hereditary disorders that severely affect skeletal muscle tissue. Skeletal muscle is indeed the most abundant tissue of the human body and it must support mechanical/physiological stress due to continuous contractions. In order to ameliorate the dystrophic phenotype the mutated gene has to be repaired/replaced into an incredibly large number of muscle fibers, whose nuclei are post-mitotic. Moreover, due to the progressive deterioration of the tissue that accompanies the progression of the disease, repairing/replacing the gene in the late phase of the disease would be very challenging and with limited efficacy outcome.
Archive Staff Only
 |
View Item |
