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Posts tagged CRISPR

ARVO 2018: Dr. Steve Rose Reports on CRISPR/Cas9 for Inherited Retinal Diseases

FFB’s own Dr. Steve Rose, chief scientific officer, reviews our commitment to funding and exploring CRISPR/Cas9 gene editing for inherited retinal disease in the video below.

FFB currently funds CRISPR/Cas9 projects at four institutions:

  • Johns Hopkins University (retinitis pigmentosa caused by the P23H mutation in RHO)
  • Columbia University (RP caused by the D190N mutation in RHO)
  • Massachusetts Eye and Ear Infirmary (RP caused by a mutation in RP1)
  • UCLA (Usher syndrome 1B caused by a mutation in MYO7A)

Want to learn more about the benefits of CRISPR/Cas9? Check out: A Cut-and-Paste Approach to Fixing Retinal-Disease Genes

Natural History Study Launches for LCA Caused by Specific Mutation in CEP290

Massachusetts Eye and Ear (MEE) is participating in a natural history study for people with Leber congenital amaurosis (LCA) type 10 caused by a mutation referred to as “c.2991+1655A>G” in intron 26 of the CEP290 gene.
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To Treat an Inherited Retinal Disease, It’s Good to Know Exactly What’s Wrong with the Gene

Jason Comander, M.D., Ph.D.In simple terms, genes are like recipes for making proteins. All the cells in our bodies “read” genetic information so they can make the critical proteins necessary to stay healthy and function properly. If there is a mistake in a gene — that is, a misspelling — a protein might not be made correctly and cells in the retina might degenerate and cause vision loss.

These misspellings are called mutations, and just like a mistake in a recipe, some mutations are more devastating than others. For example, when baking a cake, let’s say there is an error in the recipe. It incorrectly calls for a quarter cup of sugar, when the right amount is a half of a cup. The cake may not taste great, but it is still edible. But let’s say the instruction for adding flour is omitted entirely. Then the cake will be a complete failure and go uneaten.

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ARVO 2015 Highlight: A Cut-and-Paste Approach to Fixing Retinal-Disease Genes

gene editingI just returned from the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), the world’s largest eye-research conference, held this year in Denver. It attracted more than 11,000 scientists and physicians, including many of the 187 retinal researchers funded by the Foundation. The FFB science team and I worked feverishly to learn as much as possible about the latest news from the retinal-research front. It was truly exhilarating—albeit, at times, overwhelming.
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