Descrizione del progetto
Avanzati strumenti di editing genetico per una terapia genica più sicura
La terapia genica offre un approccio promettente per trattare malattie incurabili attraverso il silenziamento, l’attivazione o la ricodificazione di geni specifici. Oltre alle strategie di trasferimento genico convenzionali, le tecnologie di editing genetico di nuova concezione forniscono le potenzialità per una precisa manipolazione del genoma. Il progetto UPGRADE, finanziato dall’UE, intende affrontare determinate limitazioni che ostacolano la terapia genica associate a mutagenesi inserzionale, effetti indesiderati o efficienza limitata. I ricercatori svilupperanno nuove tecnologie rivoluzionarie per un editing genetico ed epigenetico di precisione, nonché per l’inserzione genica. In seguito a una rigorosa convalida, il progetto porterà allo sviluppo di avanzati medicinali e strumenti versatili che potrebbero essere utilizzati per svariate malattie.
Obiettivo
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.
Campo scientifico
- 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
Parole chiave
Programma(i)
Invito a presentare proposte
Vedi altri progetti per questo bandoBando secondario
H2020-SC1-2018-Single-Stage-RTD
Meccanismo di finanziamento
RIA - Research and Innovation actionCoordinatore
00185 Roma
Italia