Gene therapy has been used to restrore sight

For the first time in humans, gene therapy has been used to restrore sight. The study, at the University of Pennsylvania, was designed to test the safety of the procedure, but the results were much better than anticipated.


The treatment was applied to two patients suffering from Leber’s Congenital Amaurosis (LGA), a rare congenital condition in which the patient’s have severely reduced vision due to the lack of the RPE65 gene, a retinal pigament epithelium specific protein, which is required for the maintenance of the epithelial cells covering photoreceptor cells in the retina.


Estimates showed that light levels after 90 days were increased by a factor of almost 5 orders of magnitude compared to the pre-treated state.


However, this is a treatment for just one type of LGA, which is merely one of many forms of blindness, the causes of which are often much more comlicated in terms of their etiology compared with the current study. Nevertheless, this study demonstrates the applicability of gene therapy as a paradigm to the treatment of both congenital and degenerative forms of blindness.


Nick Rhodes

Composition of Armed Forces Institute of Regenerative Medicine

The composition of the Armed Forces Institute of Regenerative Medicine (AFIRM) has just been announced by the US Department of Defense (DoD).  Unusually, the US Army Institute for Surgical Research at San Antonio in Texas will collaborate heavily with two civilian consortia in a quarter of a billion dollar initiative to accelerate the development of regenerative technologies for treating wounded soldiers.  This has been described as the largest ever federal investment in tissue engineering and regenerative medicine.


 The initial five year programme will develop technologies in the following areas:

  • Craniofacial repair
  • Non-scarring wound repair
  • Reconstruction following burns
  • Limb reconstruction, regeneration or transplantation
  • Compartment syndrome


Of the total, only one third will be provided from military budgets, and that will be shared between the US Army Medical Research Material Command, the Office of Naval Research, the National Institutes of Health, the Air Force Office of the Surgeon General and the Department of Veterans Affairs.


The two civilian consortia are led by the Wake Forest Institute for Regenerative Medicine with the McGowan Institute for Regenerative Medicine at the University of Pittsburgh, and Rutgers University with the Cleveland Clinic.  Of note is the inclusion of UK-based tissue engineering company Intercytex established in Manchester in the development of skin.


Nick Rhodes