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Control multidimensional de la función terapéutica de las células humanas con circuitos genéticos sintéticos
Hui-Shan Li1,2, Divya V Israni1,2, Keith A Gagnon1,2
1Biological Design Center, Boston University, Boston, MA, USA.
Science (New York, N.Y.)
|December 15, 2022
Resumen
Los investigadores desarrollaron circuitos genéticos sintéticos utilizando proteínas humanas para controlar con precisión las funciones celulares para terapias genéticas avanzadas. Esta plataforma permite un control robusto in vivo de las células T para mejorar las respuestas terapéuticas.
Área de la Ciencia:
- Biología sintética
- Biología molecular y celular
- Biotecnología
Sus antecedentes:
- Las terapias génicas y celulares requieren un control preciso de la función celular humana.
- Las plataformas existentes para desarrollar circuitos genéticos sintéticos en células humanas primarias son limitadas, lo que dificulta la traducción clínica.
- Se necesitan plataformas robustas y clínicamente aplicables para diseñar circuitos genéticos sintéticos en células humanas.
Objetivo del estudio:
- Desarrollar una nueva plataforma para la ingeniería de circuitos genéticos sintéticos en células humanas primarias.
- Demostrar un control preciso y definido por el usuario sobre genes terapéuticamente relevantes utilizando estos circuitos.
- Para permitir la activación secuencial de los programas celulares para efectos terapéuticos sinérgicos.
Principales métodos:
- Desarrollo de reguladores sintéticos de la transcripción del dedo de zinc (synZiFTR) basados en proteínas derivadas del ser humano.
- Ingeniería de interruptores genéticos y circuitos para el control ortogonal utilizando inductores de moléculas pequeñas aprobados por la FDA.
- Aplicación de la plataforma en células T primarias para controlar la proliferación y la actividad antitumoral.
Principales resultados:
- Se ha demostrado un control preciso y definido por el usuario sobre los genes terapéuticamente relevantes en las células T primarias.
- Circuitos diseñados capaces de activar secuencialmente múltiples programas celulares.
- Se lograron respuestas terapéuticas sinérgicas mediante la activación coordinada de las células T.
Conclusiones:
- La plataforma synZiFTR desarrollada proporciona un método robusto para la ingeniería de circuitos genéticos sintéticos en células humanas primarias.
- Esta plataforma facilita el control preciso de las funciones celulares para terapias basadas en genes y células.
- Acelera el desarrollo y la traducción clínica de circuitos genéticos sintéticos para diversas aplicaciones terapéuticas.
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