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Posts tagged Gus Aguirre

Researchers Identify Canine Model of LCA (NPHP5) — Pursue Gene Therapy

Photo of William Beltran, Artur Cideciyan, Gustavo Aguirre and Samuel Jacobson. Photo by John Donges/Penn Vet

William Beltran, Artur Cideciyan, Gustavo Aguirre and Samuel Jacobson. Photo by John Donges/Penn Vet

When scientists embark on developing a treatment for an inherited retinal disease, one of their first tasks is to identify or create a model of the condition. Disease models can be cells in a Petri dish, a genetically engineered mouse or rat, or larger animal such as a pig. Each type of model has its pros and cons, including cost and similarity of disease characteristics to those in humans.

The investigators then use the model to study how vision is lost — that is, they figure out which types of retinal cells degenerate, what is causing the degeneration, and how quickly the cells stop working. After they gain an understanding of the disease, researchers evaluate potential therapeutic approaches using the model as a testing platform.

The goal: Move a therapy into a human study.
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Despite Blindness, the Peaches are Sweet in Paran

Ignacio, a farmer with RP, tends to his peach orchard in Peru.Paran, a village of 300 people tucked in the foothills of the Andes near Lima, Peru, is known for its sweet peaches, but also its high rate of blindness. About one in eight Paranos have lost their vision.

Until recently, the villagers never understood the cause of the affliction, though they suspected there might be a genetic component because it runs in families. However, most had never even received care from a doctor.
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