Enfermedad de Morquio A: Conclusiones

CONCLUSIONES Y PERSPECTIVAS

Durante los últimos 30 años se han realizado importantes avances en el diagnóstico y tratamiento para la enfermedad de Morquio A. En la última década, mediante la colaboración con los Drs. Shunji Tomatsu y Adriana M. Montaño, colombiana radicada en la ciudad de Saint Louis (USA), un número importante de investigadores colombianos han participado activamente en la identificación de mutaciones, evaluación de nuevas herramientas diagnósticas, construcción de modelos animales y en el desarrollo de estrategias de terapia génica y de reemplazo enzimático. Sin embargo, aún es necesario profundizar en el conocimiento de las bases moleculares de la enfermedad, el desarrollo e implementación de programas de tamizaje neonatal, el desarrollo de sistemas que permitan evitar el rechazo inmunológico de la proteína recombinante, la producción de la proteína recombinante en otros sistemas de expresión, y el desarrollo de alternativas terapéuticas basadas estrategias de reducción u optimización de sustrato o el uso de chaperonas farmacológicas. En la actualidad estas estrategias se encuentran bajo evaluación, en las cuales investigadores colombianos siguen realizando aportes importantes que permitan seguir avanzando en el conocimiento de esta enfermedad.

AGRADECIMIENTOS

Los autores expresan sus agradecimientos a la Pontificia Universidad Javeriana; la Fundación para la Promoción de la Investigación y la Tecnología del Banco de la República; el Departamento Administrativo de Ciencia, Tecnología e Innovación (Colciencias); y la Fundación Carol Ann (Organización Internacional Morquio) por sus aportes económicos para la realización de los diferentes proyectos de los cuales se derivan estos resultados. Igualmente expresan sus agradecimiento por el apoyo en la construcción de vectores para terapia génica a Felipe Garcia, Mónica Gutierrez, María Andrea Rueda, Rocio Cuaspa y Eric Shelley; en el diagnóstico y estudio de mutaciones a Hugo Vega, Olga Yaneth Echeverri, Susana Vélez, Piedad Sarmiento e Inés Stella Morales; y en la producción de la enzima recombinante en E. coli a Ángela Mosquera, Ángela Espejo, Lina Lizaraso, Alexander Rodríguez y Oscar Sánchez.

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Fecha de recibido: Junio 21 de 2011
Fecha de aprobado: Junio 30 de 2012
Dirección para correspondencia:
Carlos Javier Alméciga Díaz
e-mail: cjalmeciga@javeriana.edu.co
Luis Alejandro Barrera
e-mail: abarrera@javeriana.edu.co