Watch this Striatech Journal Club to explore a gene therapy approach targeting common oxidative stress and metabolic issues across multiple forms of blindness.
There are more than 200 human disease genes that can lead to blindness. Although gene therapy in which each disease gene is augmented or edited is possible, this approach would be extremely expensive and logistically challenging. To provide an alternative and more general approach, Cepko et al. have been analyzing mouse models of blindness, searching for problems that are common across genotypes. Studies of these mouse models led to the hypothesis that oxidative damage, metabolic deficiencies, and inflammation lead to the loss of color and daylight vision. To address these issues, many different types of genes were delivered using adeno-associated viruses (AAVs). Those that successfully prolonged vision are presented.
Key Topics Include:
- Why do cone photoreceptors die even though they don’t express the disease gene?
- Which genes can combat oxidative damage, inflammation, and glucose shortage in mouse models of retinitis pigmentosa?
- A new model of dry age-related macular degeneration.
- A potential therapy for dry age-related macular degeneration.
- Categories: Genetics & Genomics, Neuroscience, Vision Research
- Tags: age-related macular degeneration, eye disease, gene editing, gene therapy, inflammation, metabolic disease
Presenters
Constance Cepko, PhD
Professor
Genetics and Ophthalmology
Harvard Medical School
Webinar Host
Striatech
