RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

We designed a unique nanocapsule for efficient single CRISPR-Cas9 capsuling,
noninvasive brain delivery and tumor cell targeting, demonstrating an effective and safe …

CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA
ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic …

Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …

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 …

The emerging clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-
associated system (Cas) gene-editing system represents a promising tool for genome …

Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …

Clustered regularly interspersed short pallindromic repeats (CRISPR) and CRISPR
associated proteins (Cas) system (CRISPR-Cas) came into light as prokaryotic defence …