How close is clinical use of gene therapy?

How close is clinical use of gene therapy?

Panel says tissue-engineered products could be on the market within the decade

According to Regis O’Keefe, MD, PhD, biotechnologies such as gene therapy and tissue engineering are developing rapidly and could soon be part of an orthopaedic surgeon’s “toolbox.”

Regis O’Keefe, MD, PhD

“Gene therapy using stem cells is a lot closer to clinical use in orthopaedics than most people think,” said Dr. O’Keefe during a media briefing held yesterday. “These tissue-engineered products could be on the market in five to ten years.”

Johnny Huard, PhD, and Steven A. Goldstein, PhD, joined Dr. O’Keefe on the panel, which focused on the development and use of gene therapy and tissue engineering in orthopaedics. The goal of tissue engineering is to create living tissue to replace or repair diseased tissue. Tissue-engineered products for orthopaedics may facilitate repair or serve as a “functional replacement” for damaged or diseased bone, muscle or ligaments.

Among the many applications for tissue-engineered products in orthopaedics are serving as replacement for lost bone, cartilage, muscle and ligament as well as increasing or promoting bone formation in spinal fusions and with some fractures. Biological approaches are being used to improve muscle healing for sports injuries, including meniscal and ligament injuries.

The future of tissue engineering in orthopaedics is based in part on the fact that muscle stem cells are more plentiful than bone marrow stem cells. “Muscle cells have emerged as promising vehicles for gene therapy and tissue engineering in the musculoskeletal system,” said Dr. Huard. One study showed that 95 percent of muscle stem cells were incorporated into the bone.

Dr. O’Keefe pointed out that a primary reason that people go to the doctor is for issues with the musculoskeletal system. In a society where many “baby boomers” expect to stay active longer and to maintain their high quality of life, physical fitness has become a priority.

“A person cannot be fit from a cardiovascular and pulmonary standpoint without having a musculoskeletal system that is healthy and can support increased activity,” added Dr. O’Keefe. “There is a tremendous opportunity to use tissue engineering for osteoporosis, osteoarthritis, and bone repair to help keep that musculoskeletal system in good condition.”

Dr. Regis O’Keefe has an ownership stake in LaGET, Inc., a start-up company in Rochester, N.Y., that develops gene therapy to treat orthopaedic conditions.


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