Descripción del proyecto
Herramientas avanzadas de edición genética para una terapia genética más segura
La terapia genética ofrece un enfoque prometedor para tratar enfermedades incurables, por medio de silenciar, activar o recodificar genes específicos. Además de las estrategias convencionales de transferencia de genes, las tecnologías de edición genética de nueva aparición ofrecen el potencial para manipular el genoma con precisión. El proyecto UPGRADE, financiado con fondos europeos, tiene por objeto abordar ciertas limitaciones de la terapia genética relacionadas con la mutagénesis insercional, los efectos inespecíficos o la eficiencia limitada. Los investigadores desarrollarán nuevas tecnologías revolucionarias para editar genes y epigenomas con precisión, así como para insertar genes. Tras una exhaustiva validación, el proyecto dará lugar a medicamentos avanzados y herramientas versátiles, que podrían utilizarse para tratar varias enfermedades.
Objetivo
Gene therapy has recently shown remarkable potential to offer definitive treatments for otherwise incurable diseases. Currently, seven gene therapy products have reached the market and many more are entering clinical testing for selected indications. Moreover, emerging technologies for targeted gene editing are complementing the scope of conventional gene transfer, opening the way to precise gene correction and making possible to silence, activate or recode any sequence of interest in the genome. However, in order to realize the full potential of these strategies and broaden application of gene therapy, the field has to solve several major hurdles, including: risk of insertional mutagenesis by gene transfer vectors, limited efficiency and durability of some gene correction strategies, off target effects of editing tools, poor tissue targeting and immune response to editing components and delivery vehicles. UPGRADE will offer radical new solutions to overcome these hurdles. We will exploit and further develop disruptive new technologies for precision gene and epigenome editing and for site-specific transgene insertion, and stringently characterize their specificity and cellular responses. We will combine these improved technologies with advanced viral and non-viral vectors enabling cell/tissue targeting and immune evasion, to generate prototypes of advanced medicinal products (AMP). The safety and efficacy profile of each AMP will be stringently validated in tissues (hematopoiesis, heart and skeletal muscle, liver, retina) and disease models (muscle wasting, storage and blood disorders, hypercholesterolemia) paradigmatic for unmet medical need and potential long-term cure, if the limitations of current gene therapy strategies are overcome. These AMPs represent versatile products portable to the treatment of several other diseases because of related pathogenesis or correction strategies, thus providing the basis for tackling diseases affecting large patient groups.
Ámbito científico
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugs
- medical and health sciencesbasic medicineimmunology
- medical and health sciencesclinical medicineophthalmology
- medical and health sciencesclinical medicinehematology
Palabras clave
Programa(s)
Convocatoria de propuestas
Consulte otros proyectos de esta convocatoriaConvocatoria de subcontratación
H2020-SC1-2018-Single-Stage-RTD
Régimen de financiación
RIA - Research and Innovation actionCoordinador
00185 Roma
Italia