Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia
Abstract
The authors briefly describe their work in the construction of viral derived vectors for the use in gene therapy of muchopolysaccharide storage diseases (MPS), especially in Morquio A syndrome. The motivations to undertake that line of research about twenty years ago was the belief that gene therapy was the most plausible treatment for monogenic diseases due to the transient effect and its difficulty to reach bone tissue of the only effective...
Paper Details
Title
Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia
Published Date
Jul 2, 2019
Journal
Volume
27
Issue
1-2
Pages
104 - 107
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