Last month, three more patients treated with an experimental gene therapy reported improved vision. Add this to the first successful gene therapy for inherited blindness earlier this year and the science gets instant boost.
Despite years of work and resources devoted to it, experimental gene therapy had very little proven success in clinical trials. Some of the factors preventing it from effectively treating disease are the problem with viral vectors and the host’s reaction to the foreign object, in this case a virus. In 1999, a young patient participating in an experimental therapy for ornithine transcarboxylase deficiency (OTCD) died when his …read more