ProgRET will create a multidisciplinary and intersectoral European training network focusing on the mechanisms, diagnosis and therapy of dominantly inherited retinal diseases (IRD). IRD represent a major cause of blindness, affecting 350,000 people in Europe. IRD have long been considered incurable, however major advances have led to groundbreaking new treatments. Today, the most important challenges in the IRD field relate to an unsolved genetic diagnosis, unknown disease mechanisms and gene therapy development for autosomal dominant IRD (adIRD), representing 25–40% of all IRD cases. We have demonstrated an emerging role for splicing factors, structural variants and non-coding defects in patients with adIRD, and developed novel disease models and gene therapies for adIRD. ProgRET aims to dissect adIRD mechanisms using retinal stem cell and aquatic animal models, to advance adIRD diagnostics using a single-molecule multi-omics framework, and to develop innovative treatments based on RNA therapy and CRISPR-genome editing. These challenges will be tackled by integrating unique expertise and cutting-edge technology within ProgRET, including (multi-)omics, bioinformatics, functional genomics, RNA biology, gene regulation, stem cell technology, retinal organoids, animal models, genome editing and gene therapy. ProgRET will give Doctoral Candidates (DCs) unparalleled training opportunities in outstanding academic and industrial settings through training-by-research via individual research projects, secondments, and network-wide training sessions. All individual training and research activities will provide each DC with the necessary skills in academic and industrial research. ProgRET will make a career in both sectors attractive and improve their career prospects. Finally, our multidisciplinary network offers a unique opportunity to accelerate the understanding, diagnostics and therapeutics for adIRD in Europe, and to translate research findings to healthcare and society.

CRISPR genome editing technology is considered to become the greatest technological improvement in biomedical research since the invention of the polymerase chain reaction 25 years ago and pharmaceutical companies as well as academic research are eager to apply it. However, the efficiency of introducing defined changes into the genome by CRISPR is still low, currently limiting its application in basic research, industry and gene therapy. IMGENE unites expert European research groups of academia and industry to address by innovative and complementary approaches the low efficiency of precise genome editing using CRISPR technology. To our best knowledge, IMGENE is the only concerted approach to tackle the important problem of low genome editing efficiency of CRISPR in a multidisciplinary, intersectorial manner. The IMGENE consortium consisting of 6 academic and 1 industrial beneficiary (AstraZeneca), 2 industrial partners (Taconic; MilliporeSigma), and the patient organization Genetic Alliance, aims to improve the genome editing efficiency of CRISPR by research training of 8 ESRs to unleash the full potential of this technology. Combining complementary knowledge on protein chemistry, molecular biology, cellular biology, viral vectors, transgenic mice, gene therapy, and bioinformatics present in the network, IMGENE will establish novel tools and protocols for improved CRISPR genome editing efficiency that will be of immediate benefit for health and life science research, the pharmaceutical industry, and the application of gene therapy. In addition, IMGENE addresses crucial ethical questions related to the application of genome editing technology in animals, plants, and humans, which have to be solved to gain acceptance by the society. By excellent research training of 8 ESRs on a scientifically and economically highly relevant topic and additional training in transferable skills, IMGENE will educate novel leaders with great career perspectives in industry and academia.

<< Background >>Mouse-based studies are essential for deciphering the genetic basis of human diseases. However, there is a proven European and global significant deficiency of formal and specialized courses to acquire the necessary expertise in Mouse Pathobiology, especially in Higher Education. For this reason, universities, research institutions and companies, have come together to establish the PATHBIO consortium and to develop new training opportunities to change this reality.<< Objectives >>The PATHBIO objectives have been: 1. To respond to the increasing need of well-prepared professionals in the area of Mouse Pathobiology 2. To identify the main challenges for professionals working in the area of Mouse Pathobiology 3. To include new areas of knowledge and technologies in the field of Mouse Pathobiology 4. To implement an integrated European educational approach in Mouse Pathobiology<< Implementation >>The PATHBIO consortium has implemented all of the objectives envisaged in the Erasmus+ proposal, obtaining a significant impact on the target audience that has been confirmed by the external evaluation measures. Due to COVID-19 pandemic and the global restrictions, and after consultation with the Agency, PATHBIO adapted the 2020 and 2021 main events to a virtual and dual (face-to-face and on-line) formats.<< Results >>Main outputs of the PATHBIO project have been: a) Organization of three Summer Course editions with the participation of 383 students from 47 countries (28 non-European) and 60 lecturers from 13 different countries (4 non-European) b) Deployment of a Virtual Learning Platform including 270 on-line lectures c) Elaboration of 7 new teaching materials for Mouse Pathobiology d) Design of a Master program on Mouse Pathobiology submitted to the Erasmus Mundus calls in 2021 and 2022