Modified CRISPR/Cas9
Another disease was cured by editing the genome of embryos
The modified CRISPR/Cas9 genome editing method helped in an experiment on mice to cure unborn cubs from a genetic disease caused by a point mutation.
Article by Rossidis et al. In utero CRISPR-mediated therapeutic editing of metabolic genes is published in the journal Nature Medicine, a press release Guided by CRISPR, Prenatal Gene Editing Shows Proof-of-Concept in Treating Genital Disease before Birth can be read on the Penn Medicine - VM website.
More recently, with the help of genome editing, scientists from the USA have demonstrated the possibility of curing beta-thalassemia in the womb, now their colleagues have corrected the DNA of mouse embryos with another hereditary disease – tyrosinemia of the first type. The mutation in this case disrupts the exchange of the amino acid tyrosine in the body, which leads to liver and kidney damage. In order to prevent severe consequences, patients should take the drug nitizinone all their lives and follow a diet. In severe cases, they need a liver transplant.
Researchers led by William Peranteau, a specialist in pediatric and fetal surgery from the Children's Hospital of Philadelphia, used a modified genome editing method, in which only one of the two DNA helix chains is cut during the modification. Then they used specially bred mice whose genome contained a mutation similar to the one that causes tyrosinemia in humans. Like humans, mice needed nitision for life. During the experiment, 26 embryos received an injection of a virus that carried the genetic construct necessary to correct this mutation. The born mice were not given nitizion and their development was monitored. The mice from the control group developed tyrosinemia and all of them died 21 days after birth. Those whose DNA was changed successfully survived to the end of the experiment (three months).
The authors of the work admit that there is still a long way to go from the experiment on mice to the use of this method in the treatment of people. In the near future, they intend to test their method in experiments with larger animals.
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