Gene Therapy for Gyrate Atrophy

Inclusion Criteria

• •Elevated serum ornithine level greater than or equal to 200 micro M, as measured by the median of three values obtained monthly for two months prior to the screening visit and at the screening visit.
• •Absence of OAT activity measured by western blot analysis and isolated enzyme activity in skin biopsy samples.
• •Fundus findings indicative of gyrate atrophy as characterized by sharply demarcated circular patches of chorioretinal atrophy.
• •Must be at least 18 years of age.
• •Female patients of child bearing potential must have a pregnancy test done, which demonstrates a negative result and must agree to practice effective birth control for 12 months following study initiation, or until patch removal, whichever comes first.
• •Must sign and date the informed consent and is willing and able to follow study procedures.
• •Patients demonstrates progression of ocular disease due to grate atrophy evidenced by reduction in electrophysiologic testing, visual field testing or progression of retinal findings.
• •Patient has been offered an arginine-restricted diet and is unable to attain ornithine levels less than 200 micro M prior to patch placement.
• •Patient has previously undergone a skin biopsy.
• •Not currently participating in other experimental protocols or therapeutic trials.
• •Does not have a known malignancy or chronic infection yielding immunoincompetence.
• •Patient must be able to maintain follow-up and follow details of the protocol.