Projektbeschreibung
Hochmoderne Methoden zur Genomeditierung für mehr Sicherheit bei Gentherapien
Gentherapie bietet durch gezieltes Gen-Silencing, Aktivierung oder Umkodierung einen vielversprechenden Ansatz zur Behandlung unheilbarer Krankheiten. Neben den konventionellen Strategien zum Gentransfer ermöglichen die neuen Technologien zur Genomeditierung eine zielgenaue Manipulation des Genoms. Das EU-finanzierte Projekt UPGRADE will bestimmte Beschränkungen der Gentherapie nun auflösen. Dabei geht es vor allem um Insertionsmutagenese, Nebenwirkungen und begrenzte Wirksamkeit. Das Forschungsteam wird neue revolutionäre Technologien für eine präzise Genom- und Epigenomeditierung sowie für die Insertion von Genen entwickeln. Nach gründlicher Validierung werden sich aus dem Projekt hochmoderne Medizinprodukte und vielseitig einsetzbare Methoden ergeben, die bei einer Vielzahl von Erkrankungen zum Einsatz kommen können.
Ziel
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.
Wissenschaftliches Gebiet
- 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
Schlüsselbegriffe
Programm/Programme
Aufforderung zur Vorschlagseinreichung
Andere Projekte für diesen Aufruf anzeigenUnterauftrag
H2020-SC1-2018-Single-Stage-RTD
Finanzierungsplan
RIA - Research and Innovation actionKoordinator
00185 Roma
Italien