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Histological analysis of growth plate (GP) sections, active bone growth areas
.
The healthy mouse on the left and the GP of the Morquio A mouse in the middle showed severe decline in growth activity.
A research team from the University of Barcelona Autònoma de Barcelona (UAB) created the first rat model that reproduced all the disabling changes experienced by patients with mucopolysaccharidosis type IV syndrome (also known as Morquio A syndrome)
.
The team has developed a gene therapy that completely corrected the severe systemic changes in the rat model after a single intravenous injection of the viral vector
Mucopolysaccharidosis type IV syndrome is a rare disease caused by the lack of GALNS enzymes, leading to changes in bone growth (skeletal dysplasia), rapid deterioration of cartilage and complications, heart and tracheal complications leading to cardiopulmonary complications and premature death
.
The first symptoms appear in young children (approximately 2 years old), and in the most severe cases, death generally occurs around 20 years of age
UAB researchers have created the first rat model of Morquio A Syndrome.
Compared with existing mouse models, especially skeletal dysplasia, early cartilage deterioration, heart valve and tracheal changes, similar to human patients
After that, the researchers developed the first gene therapy method, which restored all the excessive pathological changes of Morquio A in the new rat model
.
This gene therapy is based on intravenous injection of a viral vector encoding the GALNS enzyme
Fatima Bosch, a researcher at UAB and the leader of the study, explained: “Gene therapy on four-week-old Morquio A rats completely restored the clinical symptoms of the disease, such as changes in bone growth, tooth deformities and fragility, Articular cartilage pathology, as well as respiratory and cardiovascular complications
.
"
Currently, there is no gene therapy method for treating Morquio A patients
.
This new treatment developed by UAB allows the enzyme to be continuously produced throughout the body, especially in the bones
"The gene therapy developed by our team may correct Morquio A in humans
.
However, before transferring the treatment to the clinical stage, it is necessary to study the biodistribution and long-term safety of the therapeutic vector in large animals
###
Nature Communications
DOI
10.
