Researchers at the London's UCL Institute of Ophthalmology have demonstrated the efficacy of gene therapy in a mouse model of LCA 1, which is caused by mutations in the GUCY2D gene, the Gucy2e-/- mouse. a dose-dependent restoration of rod and cone function and an improvement in visual behaviour of the treated mice was observed, and the effects persisted up to 6 months post injection. These excellent results prompt the investigators to propose that a similar vector may be used in a clinical trial of gene therapy for LCA1!
Read the abstract at
http://www.ncbi.nlm.nih.gov/pubmed/21671801
0 comments:
Post a Comment