Tag: Gene therapy
AAV capsid prioritization in normal and steatotic human livers maintained by machine perfusion
Therapeutic efficacy and safety of adeno-associated virus (AAV) liver gene therapy depend on capsid choice. To predict AAV capsid performance under…
The reverse transcriptase domain of prime editors contributes to DNA repair in mammalian cells
Reverse transcriptase (RT) has been shown to play a role in double-strand break repair in bacteria, yet the impact of the RT component of prime edi…
Internal cap-initiated translation for efficient protein production from circular mRNA
Circular mRNA faces challenges in enhancing its translation potential as an RNA therapeutic. Here we introduce two molecular designs that bolster c…
Evolving adeno-associated viruses for gene transfer to the kidney via cross-species cycling of capsid libraries
The difficulty of delivering genes to the kidney has limited the translation of genetic medicines, particularly for the more than 10% of the global…