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News from the Foundation
New Study Paves Way for Treating Second Eye in Gene Therapy Clinical Trial
All 12 participants in the gene therapy clinical trial for Leber congenital amaurosis (LCA) at the Children’s Hospital of Philadelphia (CHOP) have been so delighted with the vision restoration from treatment to one eye, they have been anxiously awaiting the go-ahead to have their second eye treated. Thanks to results from a Foundation-funded lab study in large animals, that will likely happen soon.
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Cell-Based Treatment Preserves Vision in Usher Syndrome Model
A research team funded by the Foundation Fighting Blindness has used cell transplantation to restore vision in a mouse model of Usher syndrome type 2A (USH2A), a leading cause of combined deafness and blindness in humans. Never before has a cell-based treatment been used to save vision in an Usher syndrome study, in large part because no other Usher syndrome animal models have exhibited vision loss or retinal degeneration. The advancement is a critical step forward in developing a vision treatment for humans with the condition.

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Foundation Develops New Strategic Plan to Guide Future Research
In keeping with its mission, and to ensure it has a focused and clinically relevant research program, the Foundation Fighting Blindness uses a strategic planning process to develop recommendations regarding long-term research goals. Held every four to five years, the most recent strategic planning meeting occurred in late 2008. A summary of the final outcomes from this effort and their implications for the next four to five years is presented here.
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