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 …

Messenger RNA is a class of promising nucleic acid therapeutics to treat a variety of
diseases, including genetic diseases. The development of a stable and efficacious mRNA …

Lipid nanoparticles (LNPs) have demonstrated efficacy and safety for mRNA vaccine
administration by intramuscular injection; however, the pulmonary delivery of mRNA …

Background: The Toll-like receptor (TLR) family is involved in the recognition of and
response to microbial infections. These receptors are expressed in leukocytes. TLR …

Fabry disease is a lysosomal storage disorder caused by the deficiency of α-galactosidase
A. Enzyme deficiency results in a progressive decline in renal and cardiac function, leading …

Diabetes is a common and important complication of cystic fibrosis, an autosomal recessive
genetic disease due to mutations in the cystic fibrosis transmembrane conductance …

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 …

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 …

Insulin-like growth factor I (IGF-I) is a growth-promoting anabolic hormone that fosters cell
growth and tissue homeostasis. IGF-I deficiency is associated with several diseases …

Synthetic biology has great potential for future therapeutic applications including
autonomous cell programming through the detection of protein signals and the production of …