American geneticists from the University of California at Berkeley have developed a method of gene therapy for sickle-shaped anemia, a rare and dangerous hereditary disease. The technology involves the use of bird DNA fragments. The study was published in the scientific journal magazine Nature Biotechnology (NatBiotech).
The method, called Precision RNA-Mediated Insertion of Transgenes (PRINT), is based on the use of retrotransposons, i.e. retrotransposons that encode RNA in the genome. PRINT allows entire genes to be inserted into the genome without causing cancer-causing mutations.
PRINT involves integrating new DNA into a cell using techniques similar to CRISPR-Cas9 genome editing technology. However, unlike CRISPR-Cas9, PRINT does not remove mutations but adds additional structures to the genome to correct the causes of genetic diseases.
Many inherited diseases, such as cystic fibrosis and hemophilia, are caused by many different mutations in the same gene, each of which disrupts the gene’s function. Any CRISPR-Cas9-based gene editing therapy needs to be tailored to a person’s specific mutation. Gene boosting using PRINT could instead deliver the correct gene to each person with the disease, allowing the patient’s body to produce the normal protein regardless of the underlying mutation.
To find a suitable retrotransposon, scientists examined the genomes of more than a dozen creatures, from insects to horseshoe crabs. Oddly enough, the best choices were found in birds – zebra finches and white-necked sparrow buntings.
According to the team, they have received confirmation that the PRINT method works. However, for its practical application, additional research on human DNA is required.
Previous scientists developed Genetic treatment of a rare hereditary disease affecting children.