Retina

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Children who develop the pediatric eye cancer retinoblastoma often lose their vision or an eye due to a lack of specific, targeted therapies and a poor molecular understanding of the cancer. Researchers at UT Southwestern and the University of Miami have discovered that a molecule – estrogen-related receptor gamma, or ESRRG – becomes hyperactive and promotes tumor cell survival in retinoblastoma.

In an announcement at the American Academy of Ophthalmology’s 2022 annual meeting at McCormick Place in Chicago, the company noted that results from Phase 1/2 MGT009 study demonstrate safety profile of investigational gene therapy botaretigene sparoparvovec (AAV-RPGR) and suggest sustained vision improvement in patients with X-linked retinitis pigmentosa.