The Cas9/guide RNA (Cas9/gRNA) system is commonly used for genome editing. mRNA
expressing Cas9 can induce innate immune responses, reducing Cas9 expression. First-…

Self-amplifying RNA vaccines may induce equivalent or more potent immune responses at
lower doses compared to non-replicating mRNA vaccines via amplified antigen expression. …

Protein supplementation therapy using in vitro-transcribed (IVT) mRNA for genetic diseases
contains huge potential as a new class of therapy. From the early ages of synthetic mRNA …

Gene therapy has always been a promising therapeutic approach for Cystic Fibrosis (CF).
However, numerous trials using DNA or viral vectors encoding the correct protein resulted in a …

Background β-Thalassemia is an inherited hematological disorder caused by mutations in
the human hemoglobin beta (HBB) gene that reduce or abrogate β-globin expression. …

The ongoing advent of genome editing with programmable nucleases, including zinc‐finger
nuclease ( ZFN ), TAL effector nuclease ( TALEN ), and Clustered Regularly Interspaced …

Background/Aims: The Polo-like kinase 1 (Plk1) inhibitor volasertib is used in the treatment
of malignancy. Volasertib is partially effective by triggering suicidal death or apoptosis of …

In vitro transcribed (IVT) mRNA therapies is a promising approach for the effective and safe
treatment of various diseases. However, IVT‐mRNA triggers immune responses due to …

Being a classic monogenic disease, gene therapy has always been a promising therapeutic
approach for Cystic Fibrosis (CF). However, numerous trials using DNA or viral vectors …

The mRNA therapeutics is a new class of medicine to treat many various diseases. However,
in vitro transcribed (IVT) mRNA triggers immune responses due to recognition by human …