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Progressive retinal atrophy (PRA) is a group of genetic diseases seen in certain breeds of dogs and, more rarely, cats. Similar to retinitis pigmentosa in humans, [1] it is characterized by the bilateral degeneration of the retina, causing progressive vision loss culminating in blindness.
In a very few breeds, such as the American Staffordshire Terrier, Old English Sheepdog, Brittany Spaniel, and Gordon Setter, symptoms do not appear until adulthood or even middle age. [13] In dogs, cerebellar abiotrophy is also usually an autosomal recessive gene, but in a few breeds, such as the English Pointer, the gene is sex-linked.
A dog with degenerative myelopathy often stands with its legs close together and may not correct an unusual foot position due to a lack of conscious proprioception. Canine degenerative myelopathy, also known as chronic degenerative radiculomyelopathy, is an incurable, progressive disease of the canine spinal cord that is similar in many ways to amyotrophic lateral sclerosis (ALS).
Collie eye anomaly (CEA) is a congenital, inherited, bilateral eye disease of dogs, which affects the retina, choroid, and sclera. It can be a mild disease or cause blindness. CEA is caused by a simple autosomal recessive gene defect. There is no treatment.
JEB is an autosomal recessive trait; both parents must carry the recessive gene in order to have an affected offspring. If N represents a normal individual and J represents an affected individual, the following crosses indicate the rate of occurrence among related horses. [6] (N/J) x (N/J) = 50% N/J, 25% N/N, and 25% J/J
Mice and dogs with MPS I have been successfully treated with gene therapy. Most vectors can correct the disease in the liver and spleen, and can correct brain effects with a high dosage. Gene therapy has improved survival, neurological, and physical symptoms; however, some animals have developed unexplained liver tumors.
Heart failure has historically been irreversible, but a new study suggests that could change. At the University of Utah, scientists used a new gene therapy that reversed heart failure in animals.
Gene therapy may be classified into two types by the type of cell it affects: somatic cell and germline gene therapy. In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete , germ cell , gametocyte , or undifferentiated stem cell .