RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

Genome editing is the modification of genomic DNA at a specific target site in a wide variety
of cell types and organisms, including insertion, deletion and replacement of DNA, resulting …

It is estimated that the human genome encodes 15% of proteins that are considered to be
disease-modifying. Only 2% of these proteins possess a druggable site that the approved …

The concepts of developing RNAs as new molecular entities for therapies have arisen again
and again since the discoveries of antisense RNAs, direct RNA-protein interactions …

Clustered randomly interspaced short palindromic repeats (CRISPRs) and its associated
endonuclease protein, ie, Cas9, have been discovered as an immune system in bacteria …

Cell membrane-coated nanoparticles (NPs) have attracted growing attention in the field of
targeted delivery strategies, which successfully combine the advantages and properties of …

Biomedical researchers have subsequently been inspired the development of new
approaches for precisely changing an organism's genomic DNA in order to investigate …

Therapeutic genome editing harnesses the power of genome editing tools to correct
erroneous genes associated with disease pathology. To bring the CRISPR/Cas9 tool from …

A single gene mutation can cause a number of human diseases that affect the quality of life.
Until the development of clustered regularly interspaced short palindromic repeats …

CRISPR/Cas, an adaptive immune system in bacteria, has been adopted as an efficient and
precise tool for site-specific gene editing with potential therapeutic opportunities. It has been …