La fibrosis quística debería ser un candidato ideal para la terapia génica por lo siguiente: 1) el defecto está en un solo gen, 2) es una condición recesiva, 3) el principal órgano afectado es el pulmón que es de fácil acceso y 4) su enfermedad progresiva con un fenotipo virtualmente normal al nacimiento, ofreciendo una ventana terapéutica.
La terapia génica se ha venido desarrollando por mas de 20 años, entre estas opciones tenemos a vectores virales en cuyo genoma se ha insertado la secuencia codificante del CFTR y vectores no virales:
Vectores virales: Genoma con gen codificante del CFTR
A continuación se presentan varias alternativas basados en vectores virales:
Se presenta un esquema de de como lo vectores virales infectan las células y liberan su genoma con el gen del CFTR
Vectores no virales
La utilización de liposomas han mostrado resultados contradictorios, en la figura se demuestra como actúan los liposomas que contienen plasmidos que codifican el CFRT
Los ensayos que han evaluado estos sistemas de liberación se demuestran a continuación en una revisión realizado por T.Lee
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1134927/
http://www.medri.uniri.hr/~zpetek/nastava/S6%20genska%20terapija/S6.6._cisticna_fibroza_2.pdf
http://www2.massgeneral.org/bbs/gen228_2009_pdfs/Griesenbach%20Gene%20transfer%20to%20the%20lung.pdf
https://www.youtube.com/watch?v=XmIas9Ccrk0
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