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Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies; however, it faces a number of unique challenges, including the need to deliver a therapeutic vector to all muscles throughout the body. In Muscle Gene Thera…
This advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy, and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations.
Today, progress in rAAV-mediated gene transfer is so robust that long-term, efficient, and regulatable transgene expression is reproducibly achieved in large animal models. The complexity of gene transfer agents in the context of their clinical use requires investigators from a …
La 4e de couverture indique : "This volume provides insight into recent developments on experimental and clinical strategies for cancer gene therapy. Gene Therapy of Solid Cancers: Methods and Protocols guides readers through protocols on gene therapeutic strategies in combinati…
This volume provides insight into recent developments on experimental and clinical strategies for cancer gene therapy. Gene Therapy of Solid Cancers: Methods and Protocols guides readers through protocols on gene therapeutic strategies in combination with helpful technical notes…
Muscle gene therapy
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Type: Book
"Muscle disease represents an important health threat to the general population. Unlike diseases affecting other tissues/organs, therapeutic options are quite limited for many inherited muscle diseases such as Duchenne muscular dystrophy. There is essentially no cure. Gene thera…
