http://splice-bio.com/the-first-ther...is-pigmentosa/
Quote:
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While a 13% success rate in correcting the mutation may sound low, with the rapid growth in gene editing technology, this preliminary success is promising for treating retinitis pigmentosa.
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This is a progressive eye disease with no cure at the moment. MY current conversation with patients, who have this, is whether they would like to test their kids or not for the gene. It typically means guaranteed blindness. This is game changing stuff.