Publications by authors named "Malte Ulrich Ritter"
Article Synopsis
- Safety is crucial in gene therapies for inherited preleukemia syndromes like severe congenital neutropenia (CN), and various CRISPR/Cas9 strategies were tested on CD34 cells from CN patients.
- All gene editing methods, including universal knockout and allele-specific mutation correction, showed at least 30% editing success without toxicity and helped restore blood cell production.
- Personalized assessments of off-target effects were conducted using patient-derived stem cells, revealing that allele-specific methods had the best safety profiles, highlighting the need for careful strategy selection in gene therapies for these diseases.
