The Journal of Bone and Joint Surgery (American) 84:1046-1061 (2002)

© 2002 The Journal of Bone and Joint Surgery, Inc.

Instructional Course Lecture

Gene Therapy in Orthopaedic Surgery

David Hannallah, MD, MSc, Brett Peterson, MD, Jay R. Lieberman, MD, Freddie H.
Fu, MD, DSci(hon), DPs(hon) andJohnny Huard, PhD
An Instructional Course Lecture, American Academy of Orthopaedic Surgeons

David Hannallah, MD, MSc

Freddie H. Fu, MD, DSci(hon), DPs(hon)
Department of Orthopaedic Surgery, University of Pittsburgh Medical Center, Liliane S. Kaufman Building,
3471 Fifth Avenue, Suite 1010, Pittsburgh, PA 15213

Brett Peterson, MD
Jay R. Lieberman, MD
Department of Orthopaedic Surgery, University of California at Los Angeles School of Medicine, Center for
the Health Sciences, Los Angeles, CA 90095-6902

Johnny Huard, PhD

Growth and Development Laboratory, Children's Hospital of Pittsburgh, 4151 Rangos Research Center,
3705 Fifth Avenue, Pittsburgh, PA 15213. E-mail address for J. Huard: jhuard@pitt.edu

Introduction
Gene therapy has the potential to revolutionize the treatment of a variety of
musculoskeletal disorders. In the last decade, more than 4000 patients have been enrolled
in clinical trials involving gene therapy. In this article, we define gene therapy and
explain why it is becoming increasingly important in modern orthopaedic clinical practice.
We also address the issues that physicians and scientists face when designing an
experiment or clinical trial involving gene therapy. Finally, we review the potential
clinical applications for gene therapy in the treatment of patients with musculoskeletal
problems.

What Is Gene Therapy?

In 1998, the Oxford Medical Dictionary defined gene therapy as "the treatment directed
to curing genetic disease by introducing normal genes into patients to overcome the effects
of defective genes . . ." 1 This definition reflects the original premise behind gene therapy,
which sprang from the belief that if a defective generesulting in a specific disease (such as
the defective fibrillin gene that causes Marfan syndrome) could be replaced with a
 home | CME | help | feedback | subscriptions |

Stanford University Libraries' HighWire Press (TM) assists in the publication of JBJS Onlin
Copyright © 2002 by The Journal of Bone and Joint Surgery, Inc. Online ISSN: 1535-138
The Journal of Bone and Joint Surgery®, JBJS®, JB&JS®, and eJBJS® are registered in
U.S. Patent and Trademark Office.