Scientists at St. Jude Children's Research Hospital have developed a tool that can locate safe places to introduce genes into human DNA. The tool is an early step in the process of improving the safety and effectiveness of gene and cell therapies. The research was recently published in the journal G
The new tool has the potential to improve gene and cell therapies.. The tool is an early step in the process of improving the safety and effectiveness of gene and cell therapies. The research was recently published in the journal“We’ve created the Google Maps of editing the genome,” said co-corresponding author Yong Cheng, Ph.D., St. Jude Department of Hematology. “With this tool, we provide a new approach to identify places to safely integrate a gene cassette.
As a result, scientists have looked for “safe harbor sites” in the genome, or locations where a gene may be introduced without causing cancer or other issues. The researchers developed a pipeline that searches for safe harbor sites using genomic and epigenetic information from specific tissues, such as blood cells.
“Our method is a new way to identify genomic safe harbor sites in a tissue-specific manner,” Cheng said. “Nobody has tried it from this angle. Our first step was to find the genomic loci that show a high frequency of insertion or deletion among healthy individuals.”
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