Researchers Develop New Method for Gene Integration in Human Cells
Scientists have developed a new method for programmable gene insertion in human cells using a laboratory-evolved CRISPR-associated transposase (CAST).
CRISPR-associated transposases (CASTs) are enzymes that can be guided by RNA to insert DNA into specific locations in the genome.
This new technique has the potential to enable mutation-agnostic treatments for loss-of-function genetic diseases, as well as facilitate various applications in the field of life sciences.
The team of researchers used directed evolution to engineer a CAST enzyme with improved activity and specificity for gene insertion in human cells.
The laboratory-evolved CAST, called CAST-IT, was able to efficiently insert DNA into targeted sites in the human genome, with a high level of precision and accuracy.
The researchers also demonstrated that CAST-IT could be used to insert large DNA sequences, up to 10 kilobases in length, into the genome.
This new method of programmable gene insertion could have significant implications for gene therapy and other genetic engineering applications.
In gene therapy, faulty genes can be replaced or repaired using this technique, potentially providing a cure for genetic diseases.
Additionally, this method could be used to insert genes that produce therapeutic proteins, such as insulin, directly into the genome of patients with genetic disorders.
The researchers also envision that this technique could be used to study the function of specific genes by inserting them into the genome and observing the effects on cellular processes.
Furthermore, this method could be used to create genetically modified cell lines for research purposes, as well as for industrial applications such as the production of biopharmaceuticals.
Overall, the development of CAST-IT provides a powerful tool for precise and efficient gene insertion in human cells, opening up new possibilities for genetic medicine and biotechnology.
Original Source: https://pubmed.ncbi.nlm.nih.gov/40373119/
