Cationic polymers have been studied as promising nonviral gene delivery vectors. In contrast to the conventional polycations with long sequences of covalently bonded repeating units, we have developed ...

The functional or regulatory processes within the cell are explicitly governed by the expression levels of a subset of its genes. Gene expression time series captures activities of individual genes ...

Before gene therapy, the median annualized bleeding rate was 14.0 episodes (IQR, 12.0 to 21.5) among all 10 participants, including 3 participants who were receiving on-demand treatment.