In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …

Prime editor (PE), a new genome editing tool, can generate all 12 possible base-to-base
conversions, insertion, and deletion of short fragment DNA. PE has the potential to correct …

Protein semisynthesis—defined herein as the assembly of a protein from a combination of
synthetic and recombinant fragments—is a burgeoning field of chemical biology that has …

Protein stability is a topic of major interest for the biotechnology, pharmaceutical and food
industries, in addition to being a daily consideration for academic researchers studying …

Cardiovascular disease remains the leading cause of morbidity and mortality in the
developed world. In recent decades, extraordinary effort has been devoted to defining the …

The prime editor (PE) can edit genomes with almost any intended changes, including all 12
possible types of base substitutions, small insertions and deletions, and their combinations …

Inteins are protein segments capable of joining adjacent residues via a peptide bond. In this
process known as protein splicing, the intein itself is not present in the final sequence, thus …