Nanoparticles deliver gene therapy that alleviates thalassemia in mice


Researchers led by Yale University have corrected a gene mutation in mice that causes thalassemia, thus alleviating symptoms of the blood disorder. This could lead to a similar gene therapy to help humans with the condition.

The team identified a protein in bone marrow that can activate stem cells. They combined the protein with synthetic molecules that act like DNA and encased the combo in nanoparticles that they delivered to mice via intravenous injection, according to a statement. The synthetic DNA, known as PNAs, bring about gene editing by binding to the target gene and prompting the cell to repair the mutation.

Thalassemia is characterized by lower-than-normal levels of oxygen-carrying hemoglobin. The researchers found that the treatment corrected the mutation in enough cells to alleviate symptoms.

Virtual Roundtable

ESMO Post Show: Highlights From the Virtual Conference

Cancer experts and pharma execs will break down the headline-making data from ESMO, sharing their insights and analysis around the conference’s most closely watched studies. This discussion will examine how groundbreaking research unveiled over the weekend will change clinical practice and prime drugs for key new indications, and panelists will fill you in on the need-to-know takeaways from oncology’s hottest fields.

"The fundamental result here is that with nanoparticles containing PNAs, along with template DNA, and simple IV infusion of molecules, we achieved enough gene editing to effectively cure the anemia in mice that had thalassemia," said Dr. Peter Glazer, a professor of therapeutic radiology and genetics at Yale.

While gene therapy is being floated as a potential treatment for blood disorders such as thalassemia, its success has so far been confined to lab dishes and not in live animals. For example, a group of physicians and researchers recently used CRISPR editing to correct the mutation that causes sickle cell disease in stem cells derived from patients. But because the Yale-led team’s approach uses synthetic DNA, it sidesteps unplanned side effects that can come with other genome-editing methods, such as CRISPR. "We demonstrated we have extremely low off-target effects," Glazer said.

Suggested Articles

Chi-Med has detailed plans to seek approval from the FDA later this year in part on the strength of data from Chinese phase 3 trial.

Takeda tapped Roche’s Foundation Medicine to develop tissue- and blood-based companion diagnostic tests for its portfolio of lung cancer therapies.

The clamor for more transparency from the leading pandemic vaccine contenders has been getting louder.