First Gene Therapy Clinical Trial to Address Genetic Blindness

PharmaNewsIntelligence | March 11, 2020

First Gene Therapy Clinical Trial to Address Genetic Blindness
Oregon Health & Science University (OHSU) held the first-ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10, according to a recent press release The clinical trial, sponsored by Allergan plc and Editas Medicine, is one of 14 different clinical trials investigating new genetic treatments for ophthalmic conditions and nearly 50 vision-related clinical trials. The procedure uses gene-editing tool CRISPR to edit human genes, also known as in vivo gene editing. Previously, gene-editing methods have edited genetic material after it was removed from the human body. Being able to edit genes inside the human body is incredibly profound,” said Mark Pennesi, MD, PhD, the Kenneth C. Swan, associate professor of ophthalmology in the OHDU School of Medicine and chief of the OSHU Casey Eye Institute’s Paul H. Casey Ophthalmic Genetics Division.

Spotlight

With the prevalence of asthma and chronic obstructive pulmonary disease (COPD) growing worldwide, the availability of primary human cells from these respiratory diseases is critical to increasing research and knowledge about the disease at a cellular level. In this study, we sought to identify genes differentially regulated in asthma and COPD lung fibroblasts (DHLF-asthma and DHLF-COPD).

Spotlight

With the prevalence of asthma and chronic obstructive pulmonary disease (COPD) growing worldwide, the availability of primary human cells from these respiratory diseases is critical to increasing research and knowledge about the disease at a cellular level. In this study, we sought to identify genes differentially regulated in asthma and COPD lung fibroblasts (DHLF-asthma and DHLF-COPD).

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