Arbor, Vol 194, No 789 (2018)

Terapias avanzadas en enfermedades raras


https://doi.org/10.3989/arbor.2018.789n3009

Cristina Fillat
Institut d’Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS) - Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), España
orcid http://orcid.org/0000-0002-0801-3338

Marcela del Río
Universidad Carlos III de Madrid - Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) - Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) - Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz (IIS-FJD), España
orcid http://orcid.org/0000-0003-2910-7189

Lucía Martínez-Santamaría
Universidad Carlos III de Madrid - Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) - Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) - Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz (IIS-FJD), España
orcid http://orcid.org/0000-0002-9637-2364

Juan A. Bueren
Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) - Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) - Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz (IIS-FJD), España
orcid http://orcid.org/0000-0002-3228-7013

Resumen


Las terapias avanzadas comprenden un grupo de medicamentos biológicos basados en la terapia génica, la terapia celular y la ingeniería de tejidos. En este artículo se presentan algunos conceptos básicos, se describen las estrategias más prometedoras y se detallan los resultados de diferentes ensayos clínicos para un grupo significativo de enfermedades, especialmente las enfermedades hereditarias monogénicas. Se hace un mayor hincapié en la terapia génica ex vivo en enfermedades del sistema hematopoyético, al ser el grupo de enfermedades pioneras que además han orientado la investigación en el campo. Destacamos también algunos ejemplos de éxito de terapia génica in vivo y señalamos la contribución de la terapia celular y de la ingeniería de tejidos al grupo de enfermedades raras de la piel. Pretendemos con ello dar una visión de la situación de las terapias avanzadas en enfermedades raras y señalamos algunas de las vías de futuro orientadas al desarrollo de tratamientos más eficaces y seguros.

Palabras clave


Terapia génica; terapia celular; ingeniería de tejidos; enfermedades raras

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