Gene therapy holds great potential as a way to treat a variety of diseases c
aused by defective genes. However, to date, a major hurdle in gene deliver
y to the correct part of the body has been the difficulty of avoiding gene o
ff-target effects (ineffective delivery). In many cases, gene drugs are encapsulated in lipid nanoparticles (liposomes), and the gene expression effici
ency of such non-viral drug carriers is usually low.
The development of lung gene therapy drugs is facing the greatest difficul
ty: (1) cannot effectively accumulate in the diseased part of the lung; (2) ca
using unnecessary exposure and Off-target toxicity. In order to drastically
improve the effectiveness and safety of lung gene therapy, our team devel
oped a new type of lung delivery nucleic acid carrier by combining differe
nt material properties, which can effectively deliver nucleic acid gene ther
apy drugs to the lungs and accumulate them during intravenous injection.
There, and using the world's first catalytic transfection effect (Catalytic tr
ansfection) to highly improve efficiency, it is the first time in the field of ge
ne delivery to propose the use of lung active targeting and catalysis to ach
ieve local target transfection targets.
We chose lung cancer as an initial disease treatment model and used nove
l vectors combined with siRNA drugs. Through preliminary cell and animal
proof-of-concept studies, we determined that this combination inhibits P
D-L1 protein expression in cancer cells. This study has confirmed that the
new vector has efficient accumulation ability in the lung and has the abilit
y of nucleic acid transfection. In an animal model of lung metastases. At th
e same time, our team is also testing different types of nucleic acid drugs,
including Plasmid DNA and Messenger RNA, etc., which have shown high
transfection activity in cell experiments, which shows that this new type of
nucleic acid carrier has a wide range of application space and value.