By injecting a therapeutic gene directly into the brain, researchers have treated a genetic disease in cats. The animals involved in the study are born with a genetic disorder directly analogous to alpha-mannosidosis (AMD).
"Through gene therapy, we replace a broken gene responsible for alpha-mannosidosis with the functioning copy, to dramatic results”, said John H. Wolfe, V.M.D., Ph.D., a neurology researcher at The Children's Hospital of Philadelphia. "The treated cats were markedly improved compared to diseased cats, with better balance and muscle control and fewer tremors.”
"In our study we could see that gene therapy used shortly after birth led to a restoration of damaged neurons, even though the lesions that represent the disease were already extensive.”
According to Charles H. Vite, D.V.M., Ph.D., assistant professor of veterinary neurology at Penn, the treated cats not only demonstrated dramatic clinical improvement, but also revealed that white matter tracks, myelin, in the brain had been largely restored.
"The ability to monitor the improvement in brain myelination in alpha-mannosidosis using imaging allows the clinician to see improvement in brain pathology without the need for brain biopsy,” Dr. Vite said.
"Lysosomal storage diseases are particularly good candidates for gene therapy because, active enzymes from genetically corrected cells will be secreted into brain tissue and taken up by neighbouring cells”, Dr. Wolfe said. "In this case we see that storage lesions throughout the brain had been greatly reduced, even though gene transfer was limited to areas surrounding where injections had been made.”
MEDICA.de; Source: Children's Hospital of Philadelphia