Resources & Publications
Recent CAROT Publications
- A CRISPR view of the 2020 Nobel Prize in Chemistry
- Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy
- Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models Oct 7, 2019
- Molecular Therapy April 13, 2019
- AAV2.7m8 is a powerful viral vector for inner ear gene therapy January 5, 2019
- SIRT1 and NRF2 Gene Transfer Mediate Distinct Neuroprotective Effects Upon Retinal Ganglion Cell Survival and Function in Experimental Optic Neuritis March 2, 2018
- Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA September 7, 2018
- Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness June 6, 2018
- https://www.jci.org/articles/view/145214
- https://www.cell.com/molecular-therapy-family/molecular-therapy/pdf/S1525-0016(20)30655-9.pdf?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1525001620306559%3Fshowall%3Dtrue
Non - Profit Partner Organizations
- ASGCT
- Foundation Fighting Blindness
- ARVO
- Choroideremia Research Foundation
- RDH12 Fund for Sight
- The Curing Retinal Blindness Foundation
- Sofia Sees Hope
- Global Foundation for Peroxisomal Disorders
- Odylia Therapeutics
Manuals
Education | Gene therapy basics
Interview with Tracy Minish - Foundation Fighting Blindness
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