Design and applications of gene therapy vectors for mucopolysaccharidosis in Colombia

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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 treatment in use, the enzyme replacement therapy. The strategy used to increase the bone targeting was to include in the vectors an aspartic acid octapeptide that increases their affinity for the oppositely charged hydroxyapatite molecule of bone. It is also discussed the difficulties to do front line research in many developing countries, due to the extended belief that their research money should be mainly devoted to projects that render solutions in a very short time. However, the authors argue in favor of doing research in gene therapy, because it is proving to be the solution for many monogenic diseases, and therefore there is a need of people with good command of GT all over the world, in order to make good use of that therapy especially for ex-vivo treatments.

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Acknowledgements

The authors are highly indebted to Pontificia Universidad Javeriana (ID PRY 3763 and 3916), Colciencias (Grant ID 120380763212 – PPTA # 8352), Fundación Banco de la República, and Saint Louis University for their support.

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Correspondence to Luis A. Barrera.

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Conflict of interest

The authors declare to be part of the patent: Delivery of Therapeutic Agents to the Bone.   Patent No.: US 7,972,593 B2; date July 5, 2011, patent that is not generating royalties.

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