gene therapy

Investigating Groundbreaking Treatments for Rare Disease

Multimedia with summary

Gene therapy has the potential to transform how we manage rare genetic diseases like hemophilia, Duchenne Muscular Dystrophy and others by addressing their root cause – genetic mutations.

The Power of Science behind gene therapy inspires our colleagues, like Bob Smith, SVP, Global Gene Therapy Business, to investigate this groundbreaking technology that could, if successful, bring disease-modification to millions of patients worldwide.

Gene Therapy for Human Disease: Clinical Advances and Challenges

Special issue of Translational Research is devoted to the topic of the gene therapy
Press Release

Philadelphia, PA, March 21, 2013 /3BL Media/ – The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today. Research articles included in the special issue highlight current studies that, after decades of trial and error, may provide evidence for a clear path of treatment and cure for many diseases. There are more than 1,800 genetic disorders known in humans, and only a small fraction of these can be treated and even fewer cured.

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