tailieunhanh - Muscle Gene Therapy

Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies. Many of these diseases are caused by a single gene mutation. While the traditional approaches may ameliorate some symptoms, the ultimate cure will depend on molecular correction of the genetic defect. The clinical feasibility of gene therapy has been recently demonstrated in treatment of a type of inherited blindness. By delivering a therapeutic gene to the retina, investigators were able to partially recover the vision in a disease once thought incurable. Compared to retinal gene therapy, muscle gene therapy faces a number of unique challenges. Muscle is one of the most. | XiXcgy 709 Springer Protocols Dongsheng Duan Editor Muscle Gene Therapy Methodsand Protocols Humana Press Methods in Molecular Biology Series Editor John M. Walker School of Life Sciences University of Hertfordshire Hatfield Hertfordshire AL10 9AB UK For other titles published in this series go to series .