Garbage genes are not nothing, it can treat heart disease
Scientists at the University of California at Los Angeles and the Howard Hughes Medical Institute have successfully used a gene that inhibits cholesterol levels as a treatment to reduce thrombocytopenia caused by familial hypercholesterolemia. In clinical pre-experiments, the researchers found that the LeXis gene lowers cholesterol and reduces arterial occlusion, and this treatment seems to reduce fat accumulation in liver cells. The study was published in the journal Circulation.
Familial hypercholesterolemia (FH) is a genetic disorder, also known as familial hyperlipoproteinemia. The clinical features are hypercholesterolemia, characteristic xanthoma, and a family history of early onset cardiovascular disease.
The LeXis gene belongs to a unique set of genes that have long been considered "junk DNA" because scientists believe they have no effect. However, evidence from the Human Genome Project indicates that genes such as LeXis are actually active. These genes are called long-chain non-coding RNAs (lncRNAs) and are one of the research hotspots in biology.
The researchers wanted to test whether a single injection of the gene LeXis could slow the progression of heart disease. To do this, they injected the mice with “LeXis†and set up a control group, then fed the mice with high-salt, high-cholesterol food for 15 weeks – the equivalent of fast food burgers and French fries. The researchers then measured the progression of heart disease.
Although previous studies have shown that IncRNAs are important, this is the first indication that IncRNAs can be used in gene therapy to treat human diseases. "Garbage genes" may help treat familial hypercholesterolemia and other difficult-to-treat diseases in the future.
The authors are Xiaohui Wu, Zhengyi Zhang, Dr. Tamer Sallam from the University of California, Los Angeles, Dr. Peter Tontonoz from the Howard Hughes Medical Institute, Dr. Marius Jones and Dr. David Salisbury.
They will next confirm this finding in large animals and test the therapy in conjunction with currently available treatments.
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