The study has revealed how to reactivate with drugs a defence mechanism that prevents the toxic accumulation of proteins in the cells.

A study by the Spanish National Research Council (CSIC) has discovered a molecular mechanism that suggests a new therapeutic strategy for treating retinitis pigmentosa, a type of retinal degeneration that affects one in 4,000 people. The work, carried out in rodents, has been published in the journal Nature Communications.

TDB keeps you informed. Follow us on Facebook, Twitter and Instagram.

The team led by researcher Patricia Boya, from the Margarita Salas Biological Research Centre (CIBMS-CSIC), has discovered how to use drugs to activate a cellular process known as chaperone-mediated autophagy (AMC). This is an essential mechanism in cellular defence against toxicity due to the accumulation of damaged proteins (a frequent problem in neurodegenerative diseases such as Parkinson’s or Alzheimer’s) but whose activity decreases with age.

“Reducing the decline of chaperone-mediated autophagy has been shown to be beneficial in experimental genetic models. In this case, we have used mice with retinal degeneration to study retinitis pigmentosa, a devastating and incurable condition that causes blindness. We have seen that the administration of AMC activators, either systemically or locally by injecting it into the eye, effectively reduces retinal degeneration and preserves sight,” explains Boya.

This study is the result of an international collaboration with the Albert Einstein College of Medicine (USA), the University of Alcalá (UAH) and the Ramón y Cajal Institute for Health Research (IRYCIS).