Scientists from Boston Children’s Hospital are investigating a gene therapy treatment to treat Usher syndrome, a genetic condition that causes deafness and blindness.
Normally when studying disorders that cause blindness in humans, scientists genetically disable cone-related genes in small animals like mice. Studying nine-banded armadillos would give scientists a much more realistic model to test viable treatment options, such as gene therapy, a method of correcting a genetic disease by replacing defective genes with corrected copies. If gene therapy were able to correct the nine-banded armadillo’s cone-related mutations, it could be adapted to correct forms of human blindness.
Scientists found that people who have night blindness also have proteins that do not function properly, and fail to send electrical images from the rods to the brain. This connection between night blindness in horses and humans gives us the opportunity to further investigate the cause of CSNB and how we can treat it in both humans and animals.
Now that Siamese cats are available as genetic models for Niemann-Pick disease, in-depth studies on the biochemical properties of the disease are being performed. Previously, studies on Niemann-Pick disease were performed on rodents with drug-induced enzyme deficiencies, which was less than ideal. Although there are obstacles to be faced, researchers are optimistic that Siamese cats will speed the search for a cure or effective treatments for Niemann-Pick disease.
Cancers involve out-of-control cell growth. Scientists believe that the zebrafish may hold the key to gaining a better understanding of how cancer moves and changes, which could provide insight into how to create better treatments. Researchers at Boston Children’s Hospital have developed a transparent mutant zebrafish commonly known as the “Casper” zebrafish that allows scientists to watch cancers develop in adult fish.