ESGCT 30th ANNUAL CONGRESS BRUSSELS 2023

Oral presentation: 

''MILESTONE, A universal CRISPR/Cas9n-mediated genome editing strategy for ELANE-related severe congenital neutropenia''. Massoud Nasri, University Hospital Tübingen

Poster presentations:

''A comparison of different strategies for CRISPR/Cas9-based gene therapies of congenital neutropenia associated with ELANE mutations''. Malte Ritter, GRTC Tübingen  

''Innovative single gRNA enhanced-deletion genome editing-based correction of aberrant splicing due to pathogenic deep-intronic variants'' Pietro De Angeli, University Clinics Tübingen, Institute for Ophthalmic Research 

''AAV-based gene therapy for hereditary spastic paraplegia type 5''. Linus Wiora, German Center for Neurodegenerative Diseases (DZNE), Tübingen, Germany 

''Chemical optimization of miR-146a mimic for the treatment of Graft versus Host disease''. Xavier Segarra Visent, University Hospital of Tübingen