[Construction of an adenoassociated, viral derived, expression vector to correct the genetic defect in Morquio A disease].

Introduction: Mucopolysaccharidosis IVA (Morquio A) is caused by a deficiency of N-acetylgalactosamine-6-sulphate-sulphatase, a lysosomal enzyme required for the stepwise degradation of keratan-sulfate and chondroitin-6-sulfate. A deficiency in this enzyme results in an accumulation of glycosaminoglycans in several tissues. Currently, no effective therapies exist and only supportive measures are used to treat some manifestations of the disease.