All Leber Congenital Amaurosis articles
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NewsGene therapy trial facilitates major vision improvement
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
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ArticleModifier gene therapy - clinical development and manufacturing considerations
Expanding on his earlier podcast discussion with EPR, Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen, discusses the company’s promising modifier gene therapy candidates for ophthalmic disorders.
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News“Landmark” genetic blindness CRISPR trial outcomes released
A Phase I/II trial has shown that a CRISPR-based gene editing therapy can be safely delivered to the retina and provide clinically meaningful outcomes.