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 …

Delivery of genetic information to the interior of target cells in vivo has been a major
challenge facing gene therapies. This barrier is now being overcome, owing in part to …

The novel coronavirus disease (COVID-19) pandemic remains a global public health crisis,
presenting a broad range of challenges. To help address some of the main problems, the …

CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

RNA therapeutics show a significant breakthrough for the treatment of otherwise incurable
diseases and genetic disorders by regulating disease-related gene expression. The …

The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

Our ability to edit genomes lags behind our capacity to sequence them, but the growing
understanding of CRISPR biology and its application to genome, epigenome and …

Abstract mRNA-based drugs have tremendous potential as clinical treatments, however, a
major challenge in realizing this drug class will promise to develop methods for safely …

Lipid‐based nanoparticles (LBNPs) are currently the most promising vehicles for nucleic
acid drug (NAD) delivery. Although their clinical applications have achieved success, the …

Ionizable lipid‐based nanoparticles (LNPs) are the most advanced non‐viral drug delivery
systems for RNA therapeutics and vaccines. However, cell type‐specific, extrahepatic mRNA …