Scientists at the Salk Institute have developed a new method for anti-aging therapy based on genetic processing using the CRISPR system. This method helps alleviate the symptoms of progeria, an incurable genetic disease that leads to premature death. This is reported in a press release about MedicalXpress.
The researchers used CRISPR / Cas9 in a murine progeria model and the animals were modified so that the Cas9 protein was synthesized in their cells. This compound is a "scissors" and cuts the DNA in a certain part of it. With the help of an adeno-associated virus, two synthetic guide RNAs involved in the process of gene processing and a reporter gene were delivered to the rodents, making it possible to determine whether the operation was successful.
Materials on the subject
Guide RNA indicated to Cas9 which gene to make a cut. The target of the system is the LMNA gene, which produces protein lamin A. A. A defective laminate A, called progerine, breaks the binding of chromosomes to the nuclear membrane, leading to the symptoms of progeria. As a result of gene therapy, LMNA became non-functional, but at the same time normal laminate C was synthesized.
Two months after the start of therapy, the condition of the cardiovascular system improved, the degeneration of the blood vessels slowed and the symptoms of bradycardia disappeared, and the life expectancy of rodents increased by 25 percent. Researchers are convinced that improved therapy will increase the range of tissues exposed to the CRISPR system and will further increase life expectancy.
The CRISPR / Cas system is used to change the nucleotide sequence of DNA. Cas is a protein that cuts a double chain on a specific site. This site determines the guide RNA (sgRNA), which is associated with a specific recognition site according to the principle of complementarity. Different sgRNAs are encoded by spacers – DNA segments located within the CRISPR – a group of special repeating sequences.