Gene Therapy Book by MDA Grantee

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MDA grantee Dongsheng Duan has published a book on the latest advances in gene therapy for muscle disease, particularly muscular dystrophies.

Duan, a professor of microbiology and immunology at the University of Missouri, served as editor of the book Muscle Gene Therapy and co-authored two of its 15 chapters. Duan is currently researching gene transfer therapy in dogs.

About the new book

Muscle Gene Therapy brings together work by numerous current and former MDA research grantees who are experts in the fields of muscle gene modification, augmentation and transfer.

Several chapters address the promise of exon skipping, a process to repair the defective gene which has proven effective in animal models and is now being tried in boys with Duchenne muscular dystrophy (DMD). Other chapters examine RNA interference, stem cell therapies, and the development and application of the muscle gene delivery vehicle known as adeno-associated viral vector, or AAV.

The book looks at special issues, including gene therapy for cardiac and respiratory muscles and gene therapy directed at babies in the womb. It also examines the bumpy road that clinical gene therapy has traveled and finds that, despite the setbacks, “muscle gene therapy has finally come of age.”

In addition to editing the book and writing the prologue, Duan co-authored chapters on Duchenne cardiomyopathy gene therapy and delivery of large therapeutic genes. Another current MDA grantee, Jeffrey Chamberlain of the University of Washington, Seattle, co-authored a chapter on systemic gene delivery.

Other contributors who have received MDA support include Barry Byrne of the University of Florida in Gainesville; Paul Gregorevic of the Baker IDI Heart & Diabetes Institute in Melbourne, Australia; Xiao Xiao of the University of North Carolina-Chapel Hill; Paula Clemens of the University of Pittsburgh; and Qi Long Lu of Carolinas Medical Center in Charlotte, N.C.

Although Muscle Gene Therapy is directed at researchers and academics, it may interest patients, families and caregivers wishing to learn more about this promising but complex field. Published by Springer in January, it is available for $179, online or in book stores.