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Posts tagged choroideremia

Choroideremia Gene Therapy Improves Vision in Human Study

Dr. Robert MacLaren, lead investigator in the gene therapy trial.The development of retinal-disease gene therapies took a monumental step forward on January 16 with a report in The Lancet that researchers were able to improve vision in five of six people with choroideremia participating in a clinical trial.
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Getting the Right Diagnosis for a Retinal Disease

Research at the monitorDefinitive diagnoses for inherited retinal diseases don’t always come easy, even for the patients of the most knowledgeable doctors. Comments posted to this blog over the past year are a testament to that fact. Many readers are understandably frustrated by a doctor’s inability to determine exactly what retinal condition is affecting them or loved ones.
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ARVO Update: News on Gene Therapy Clinical Trials for RP and Choroideremia

Dr. Robert MacLaren

Dr. Robert MacLaren

I was very excited to learn here at ARVO that two early-stage gene therapy clinical trials – one for choroideremia taking place in the U.K., the other for autosomal recessive retinitis pigmentosa (arRP) caused by MERTK mutations underway in Saudi Arabia – are proceeding well thus far. It is important to keep in mind that safety is the primary focus in these Phase I studies.
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