What should a clinician know to be prepared for the advent of treatment of retinal dystrophies?
It is now evident that several forms of therapy have influenced inherited retinal degeneration in animals. These are gene therapy, cell transplantation, slowing of cell death using growth factors and pharmacological approaches. There are firm proposals to initiate gene therapy in the foreseeable future. For therapy to be successful a variety of attributes of disease must be established so that the full benefits of research can be realised in clinical practice. These can be considered as identification of the causative genes, knowledge of the disease mechanisms and detection of the therapeutic effect.
Bird
Alan C
AC
Department of Clinical Ophthalmology, Moorfields Eye Hospital, City Road, London EC1V 2PD, UK.
D016923:Cell Death / Q000502:physiology; D015316:Genetic Therapy; D006801:Humans; D009154:Mutation; D012164:Retinal Diseases / Q000235:genetics / Q000378:metabolism / Q000628:therapy*