In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are
currently in clinical development, gene therapy holds the promise of a lasting cure with a …

Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …

Prime editing is a versatile genome editing tool but requires experimental optimization of the
prime editing guide RNA (pegRNA) to achieve high editing efficiency. Here we conducted a …

CRISPR–Cas-based genome editing holds great promise for targeting genetic disorders,
including inborn errors of hepatocyte metabolism. Precise correction of disease-causing …

Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous
factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for …

Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Gene therapy is a promising modality for the treatment of inherited and acquired
cardiovascular diseases. The identification of the molecular pathways involved in the …

CRISPR–Cas9 genome engineering is a powerful technology for correcting genetic
diseases. However, the targeting range of Cas9 proteins is limited by their requirement for a …

Phenylketonuria (PKU) due to recessively inherited phenylalanine hydroxylase (PAH)
deficiency results in hyperphenylalaninemia, which is toxic to the central nervous system …