Atlanta Journal ConstitutionFDA may approve gene therapy that could cure hereditary blindnessAtlanta Journal ConstitutionThe procedure tackles Leber congenital amaurosis, or LCA, a condition caused by flaws in the RPR65 gene, the FDA briefing documents… Continue reading
Atlanta Journal ConstitutionFDA may approve gene therapy that could cure hereditary blindnessAtlanta Journal ConstitutionThe procedure tackles Leber congenital amaurosis, or LCA, a condition caused by flaws in the RPR65 gene, the FDA briefing documents… Continue reading
Spectrum |
‘Mini-gene‘ therapy relieves features of Rett syndrome in mice
Spectrum The findings bolster the notion that gene therapy might ease features of Rett syndrome in people with the condition, says lead researcher Adrian Bird, Buchanan Professor of Genetics at the University of Edinburgh in Scotland. “Gene therapy has gone … |
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With GMO Struggle In Mind, Food Industry Vying For Public Trust With CRISPR Discussion
KUNC The technology is called Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR. It’s a technique that Alison Van Eenennaam, an animal genetics professor at University of California, Davis, says can de-activate a gene. Or, as she puts it … |
Rare Disease Report |
Part 3 of Gene Therapy Trial for MPS IIIA (Sanfilippo A) Has Begun
Rare Disease Report On Monday, Rare Disease Report covered the release of new information from Abeona Therapeutics’ gene therapy trial for patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome). One year data from Cohort 1 in the company’s 3-cohort, … |