Swimming Against the Nucleotides
Advances in cell and gene therapy have led to a spate of novel treatments for inherited retinal disease. But with each breakthrough comes new dilemmas...
James Bainbridge |
Tomorrow’s cures for retinopathies will likely include advanced gene and cell therapies delivered by precision surgery.
Translational research often requires both scientific and clinical input – and this demands collaboration between groups with complementary skill-sets. Developing gene or cell therapies for retinal disease requires sophisticated expertise in a range of disciplines from molecular biology to microsurgery. Close collaboration was critical for the 2017 approval of a gene therapy for Leber Congenital Amaurosis (LCA), which is a form of childhood blindness that can be caused by the lack of a gene called RPE65. The contribution of our team at UCL/Moorfields was recognized last year, when the 2018 Antonio Champalimaud Vision Award was awarded to the four groups working to develop gene therapy for this condition. Working on a new therapy for an unmet clinical need is its own reward – but it’s certainly an honor to receive such recognition!
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