Rescue of the endogenous FVIII expression in hemophilia A mice using CRISPR-Cas9 mRNA LNPs

• मुख्य लेखकों: Chun-Yu Chen (लेखक), Xiaohe Cai (लेखक), Barbara A. Konkle (लेखक), Carol H. Miao (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2024-12-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

Gene editing provides a promising alternative approach that may achieve long-term FVIII expression for hemophilia A (HemA) treatment. In this study, we investigated in vivo correction of a mutant factor VIII (FVIII) gene in HemA mice. We first developed MC3-based LNPs for efficient mRNA delivery int...