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 …

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

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 …

Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent
and treat various diseases. To function in vivo, mRNA requires safe, effective and stable …

mRNA vaccines have been demonstrated as a powerful alternative to traditional
conventional vaccines because of their high potency, safety and efficacy, capacity for rapid …

Abstract mRNA vaccines have evolved from being a mere curiosity to emerging as COVID-
19 vaccine front-runners. Recent advancements in the field of RNA technology, vaccinology …

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 …

In 2021, mRNA vaccines against COVID-19 were approved by the Food and Drug
Administration. mRNA vaccines are important for preventing severe COVID-19 and returning …

Loss-of-function mutations in Angiopoietin-like 3 (Angptl3) are associated with lowered
blood lipid levels, making Angptl3 an attractive therapeutic target for the treatment of human …

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 …