A research team from the Universitat Autònoma de Barcelona (UAB) has generated the first rat model recapitulating all disabling alterations experienced by patients with Mucopolysaccharidosis type IVA, also known as Morquio A disease. The team has developed a gene therapy that fully corrects the severe whole-body alterations in the rat model after a single intravenous viral vector administration. The research opens the door to a future therapy which could be administered at young ages in patients diagnosed with this rare disease, thereby preventing bone malformations, osteoarthritic complications and other life-threatening alterations.
Click here for original story, Treatment with gene therapy of a new rat model of Morquio A
Source: Phys.org