Ocular gene therapy: the basic science and current state of research.
Recent technical advances have led to the demonstration of the molecular basis of many genetic eye diseases. Methods now in use have been able to both suppress and insert genes in vitro and in vivo in ocular cells. The combination of this new knowledge and these techniques offers the potential for a new therapeutic approach to diseases that currently have no treatments. The scientific basis of gene therapy is introduced and then the current state of research is discussed in relation to the eye and ocular tissues.
da Cruz
L
L
Lions Eye Institute, Nedlands, Australia. lyndondc@cyllene.uwa.edu.au
Rakoczy
P
P
Constable
I
I
D000260:Adenoviruses, Human / Q000235:genetics; D000818:Animals; D015785:Eye Diseases, Hereditary / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods; D005822:Genetic Vectors; D006801:Humans