It is a far more serious situation if there is only one life - shortening inherited disease in a breed, but nearly all of the dogs of that breed either carry the disease gene or show symptoms of it, than if there are several less
severe inherited diseases in the breed and the disease producing genes are rare for all of these diseases.
For the first time, researchers have infused a person's blood with gene - editing tools, aiming to treat
his severe inherited disease, The Associated Press (AP) reported today.
Not exact matches
Though this research focused on the form of the
disease called Leber congenital amaurosis 2, or LCA2, the scientists and engineers involved in the study believe the technology holds promise for other forms of LCA as well as other
inherited diseases that lead to
severe vision loss or blindness.
People with the rare
inherited disease also have frequent nosebleeds, as many as seven a day, that can become so
severe that patients require skin grafts in their noses or even more desperate measures to lead normal lives.
GM1 gangliosidosis, or Landing
disease, is a rare
inherited neurodegenerative lysosomal storage disorder characterized by
severe cognitive and motor developmental delays resulting in the death of most patients at a very young age.
Advanced Cell Technology, of Marlborough, Mass., said it would test its stem cell therapy on 12 adults with
severe vision loss caused by Stargardt's, an
inherited disease.
Cataracts can also be caused by uveitis (internal eye inflammation),
severe trauma, or
inherited retinal
diseases like Progressive Retinal Atrophy.
Canine hip dysplasia is a
severe,
inherited joint
disease of dogs that reveals itself in a progressive lameness associated with arthritis and looseness of both hip joints that make running, walking, and even standing difficult.
The most serious
disease in Rhodesian Ridgeback puppies (with about a 5 % incidence) is a
severe inherited skin deformity called dermoid sinus.
Except in cases where
severe joint trauma is associated with OCD, the
disease should be presumed to be
inherited.
As well as the respiratory problems these animals suffer
severe dental problems because their jaws and teeth are deformed, they may struggle to pick up and eat food, have exposed eyes that are prone to injury, skin folds on their face that cause inflammation, infection and sometimes trauma to their eyes, spinal problems in the dogs bred for a curly tail as well as
inherited diseases due to the narrow gene pool.
Alachua, Fla. - based AGTC is a clinical - stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with
severe inherited orphan
diseases in ophthalmology.