Viral gene therapy is being tested as treatment for numerous diseases, including cancer, cystic fibrosis, heart disease, muscle diseases, diabetes and neurodegenerative disease like Parkinson's.
While gene therapy is still available only as part of a clinical trial, scientists, clinicians and families hold hope that it will eventually become a fundamental part of modern medicine and provide treatment for the currently untreatable.
Modified viruses used to deliver genetic material into cells, known as viral vectors, have become the go-to delivery capsule in gene therapy. Investigators use viral vectors to efficiently target certain cells and deliver genetic material into the cell nucleus where native genes reside.
Nationwide Children's has a unique facility that specializes in creating these viral vectors - a rare, incredibly beneficial asset to our Research Institute - called the Clinical Manufacturing Cleanroom Facility. So far, the Cleanroom Facility has produced several vectors for use in investigational new drug studies, and products for phase I clinical trials for the congenital blindness disorder, choroideremia, muscular dystrophy and inclusion body myositis. Additional trials focused on spinal muscular atrophy and Duchenne and limb girdle muscular dystrophies are in the works.
Having this facility on-site allows for expedited clinical trials at a much more affordable price, which all comes back to being able to offer the best possible care for all of our patients.
To learn more about the Clinical Manufacturing Cleanroom Facility, visit:
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