New treatment target holds potential for RP

A study published in Cell Reports in Septber identified what was said to be a potential treatment target for blinding diseases such as retinitis pigmentosa (RP) and advanced dry age-related macular degeneration.“We believe we have uncovered a unifying pathway involved in inflicting severe damage to and even causing the death of rods and cones,” co-first author Dr Jonathan Lin stated. “These findings should help us develop treatments for retinal disorders, regardless of what’s causing th.”In a series of experiments first in mice and later in retinal cells, the researchers identified a key molecule, NAD, in the process that led to the death of the retina’s rods and cones.{{quote-A:R-W:450-I:2-Q: These findings should help us develop treatments for retinal disorders, regardless of what’s causing th. -WHO:Dr Jonathan Lin, Physician at Washington University School of Medicine in St Louis}}The researchers identified probls in the photoreceptor cells’ mitochondria, where energy is produced, as a factor in vision loss. The NAD molecule was said to be known for its important role in energy production, and recent research reportedly identified gene mutations along the NAD pathway as having a role in a disease that caused babies to go blind. The team found that defects in the same NAD pathway appeared to be involved in retinal diseases as well.When they treated damaged photoreceptor cells in mice with a second molecule called NMN – a precursor molecule that boosts levels of NAD – the cells’ degeneration ceased and vision was restored.“Deficiencies in the energy pathway led to probls, including damage to rods and cones and even photoreceptor death,” senior investigator Dr Rajendra Apte explained. “This is exciting because we may have found a reason why these highly metabolically active cells are susceptible to damage and death when the NAD pathway does not function optimally.”The pathway could be a potential target for therapies for multiple retinal diseases. However, Dr Apte said he was most excited about what it might mean for conditions such as RP.“It’s one of the leading causes of blindness, and it harms vision more slowly than many other retinal diseases, causing vision loss over the course of many years,” he said. “That gives us a much larger therapeutic window.”He noted that there was potential to make a big difference in patients’ quality of life as there were currently no treatments. In addition, since scientists have already begun experimenting with the NAD pathway, with some planning to test treatments involving the NMN molecule, Dr Apte said he expected clinical human trials could begin soon.

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