Nano "rockets" escort genetic drugs to the lesions

The "launch vehicle" is ready to go.

In recent years, researchers have been trying to build efficient, safe and low-cost polymer carriers, which will give the wings of gene therapy, a high-profile medical technology, to take off

Academician Chen Xuesi of Changchun Institute of Applied Chemistry, Chinese Academy of Sciences recognized the importance and urgency of developing high-performance polymer gene carriers as early as 20 years ago

The achievement of this award is the reward of 20 years of hard work by the researchers of the institute

Gene therapy faces 'ammunition' transportation challenges

The essence of gene therapy is to achieve the purpose of treatment by regulating genes

Since the birth of gene therapy technology in the 1970s, it has been regarded as a revolution in medicine and pharmacy, which promises to cure various diseases caused by genetic defects or abnormalities

Due to its outstanding performance, gene therapy has attracted the attention of scientists around the world

But such a miraculous gene therapy faces a key problem: how to deliver the gene or drug to the target

"The human body's environment is more complex than the atmosphere.

Polymer carrier ensures delivery efficiency and safety

For gene therapy, the ideal carrier should not only be able to carry a sufficient amount of gene or drug "munitions", but also be non-toxic and pathogenic to the human body to ensure safety; it should be strong enough to protect the "munitions" from The destruction of nucleases, etc.

Polymer carriers are generally cationic polymers, which have the advantages of safety, low toxicity, easy functionalization, and mass production

Tian Huayu led the researchers to adopt the strategy of "introducing multiple interactions and synergies", grafting p-toluenesulfonyl-protected arginine on polylysine to obtain a high-performance polymer carrier, allowing electrostatic interaction, The synergy of hydrogen bond interaction and hydrophobic interaction makes the driving force for the carrier to enter the cell from single to multiple, which greatly increases the efficiency of the carrier entering the cell

"This is like upgrading a rocket.

What's more interesting is that the researchers also designed an "invisibility cloak" for this nano "rocket"

"The cationic polymer itself has a positive charge, and we hope to use the excess positive charge to help it enter tumor cells, but during transport, it can easily interact with negatively charged proteins in body fluids, or interact with non-target tissue cells Causes non-specific phagocytosis, which seriously affects the transmission efficiency of complex nanoparticles to target tissues

Therefore, the researchers cleverly wrapped the "rocket" tightly with dialdehyde-based polyethylene glycol and compressed its volume so that it could fool the negatively charged proteins in the body and enter tumor cells efficiently

The carrier is upgraded from a single function to a multi-function

From the practical effect, this new type of polymer carrier realizes the efficient transfer of gene material in vivo, and has achieved exciting results in anti-tumor therapy
.
The experimental results show that using this method, the tumor cell endocytosis efficiency and tumor growth inhibition rate are significantly improved
.

Not only that, researchers have also introduced detection and combined therapy units into the carrier to build a multifunctional carrier, which improves the ability to monitor and treat diseases in real time
.
"The polymer carrier system we constructed has the ability of gene and photoacoustic dual-mode imaging, and the function of gene and photothermal combined therapy
.
The above characteristics have upgraded the polymer carrier from a single function to a multi-function, with integrated diagnosis and treatment of tumor tissue and double killing.
ability
.
" Tian Huayu said
.

The polymer carrier combined with the shielding system has become a universal carrier platform after carrying genetic material, protein, and chemotherapy drugs
.
Using this platform, Tian Huayu et al.
further constructed a nano-drug delivery system based on two tumor acidic microenvironment responses - a chemotherapeutic drug loading system and a gene loading system, and then developed a new type of immune cocktail therapy
.
The therapy has demonstrated excellent therapeutic efficacy in several tumor models
.

Localization of gene transfection reagents breaks foreign monopoly

Related Links

Since Chen Xuesi laid out efficient gene therapy vectors 20 years ago, industrialization and commercialization have been one of the main goals of this technology
.
Although it will take some time for gene carriers to move towards clinical treatment, the time for industrialization of gene transfection reagents is ripe
.

As a key link in gene therapy, the core technology of gene transfection reagents has always been monopolized by foreign companies
.

In view of this, under the encouragement and leadership of Chen Xuesi, with the strong support of the Ministry of Science and Technology, the National Natural Science Foundation of China, the Chinese Academy of Sciences and the Jilin Provincial Department of Science and Technology, researcher Tian Huayu has been devoted to the research and development of gene transfection reagents for 20 years.
In the work, on the basis of predecessors' research, he is determined to innovate and overcome difficulties, completely getting rid of the difficult predicament that our country is controlled by others in this field
.

"The breakthrough of domestic gene transfection reagents has changed China's current situation
of relying on imports of gene transfection reagents," Tian Huayu said
.
At present, the research team has successfully developed 3 varieties of gene transfection reagents, which are gradually expanding the market and have been used in more than 50 universities, hospitals and scientific research institutions
.
(Reporter Yang Lun)