![]() ![]() Each study visit will last about 4-8 hours and will include vision function tests, dilated eye exams, and blood draws. Participation will last for at least one year with an option for long-term follow-up. NM206933.4(USH2A):c.2546G>A (p. Usher Syndrome Type 1J/ DFNB48, Usher Syndrome Type 3A/ Retinitis Pigmentosa, Usher Type 1C / DFNA18, Usher Type 1G, Usher Type 2A, Usher Type 2D /. If the participant is eligible, the gene therapy will be given by a surgical procedure to one eye within one month of the screening visit.įollowing surgery, subjects will have eight more visits at the Casey Eye Institute. Interested participants will come to the Casey Eye Institute in Portland, OR, for a screening visit that will last 2-3 days. This can be determined by visual field testing. Legal blindness as defined by peripheral (side) vision being no greater than 20 degrees.Genetic testing must also be completed within the subject's family (one or both parents or siblings) to confirm the results of the affected subject. The MYO7A gene has been linked to Usher Syndrome Type 1B. GARD: Usher syndrome type 2A is a genetic condition characterized by hearing loss from birth and progressive vision loss that usually begins in adolescence. Must have two documented mutations (changes) in the MYO7A gene that are disease-causing.The eye with worse vision will receive the gene therapy. The gene therapy is given by surgical injection underneath the retina of one eye. The study investigators want to find out if UshStat is safe for use in humans. The USH2A gene is mutated in patients with Usher syndrome type IIa, which is the most common subtype of Usher syndrome and is characterized by hearing loss and retinitis pigmentosa. The condition is clinically and genetically heterogeneous, with no current treatment. Usher syndrome refers to a group of genetically and clinically heterogeneous autosomal recessive diseases with retinitis pigmentosa (RP) and hearing. This is the first study that aims to treat RP due to Usher Syndrome by gene therapy. Usher Syndrome is the commonest cause of inherited blindness and deafness. The purpose of this study is to learn about a new gene therapy being studied in patients with Retinitis Pigmentosa (RP) as a result of Usher Syndrome. A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected UshStat®, Administered to Patients with Retinitis Pigmentosa Associated with Usher Syndrome Type 1B. ![]()
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