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
Therapy: Methods and Protocols expert researchers in the field present a collection of
techniques aimed at bridging the translational gap in muscle gene therapy between the prevalent
rodent models and vitally important larger animal models. Divided into three sections this
volume examines basic protocols for optimizing the muscle gene expression cassette and for
evaluating the therapeutic outcomes new developments in muscle gene therapy technology such as
adeno-associated viral vector (AAV) oligonucleotide-mediated exon-skipping and novel
RNA-based strategies and step-by-step guidance on muscle gene delivery in swine ovine canine
and non-human primates. Written in the highly successful Methods in Molecular Biology(TM)
series format chapters include introductions to their respective topics lists of the
necessary materials and reagents detailed readily reproducible laboratory protocols and tips
on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge Muscle Gene
Therapy: Methods and Protocols serves as an invaluable resource for graduate students
post-doctoral fellows and principle investigators pursuing the crucial advancement of muscle
disease gene therapy in the hope of someday curing these debilitating disorders.
