Researchers develop a computational approach to identify genomic safe harbors for therapeutic genes

Many future gene and cell therapies to treat diseases like cancer, rare genetic and other conditions could be enhanced in their efficacy, persistence, and predictability by so-called “genomic safe harbors (GSHs).” These are landing sites in the human genome able to safely accommodate new therapeutic genes without causing other, unintended changes in a cell’s genome that could pose a risk to patients.

However, finding GSHs with potential for clinical translation has been as difficult as finding a lunar landing site for a spacecraft -; which has to be in smooth and approachable territory, not too steep and surrounded by large hills or cliffs, provide good visibility, and enable a safe return. A GSH, similarly, needs to be accessible by genome editing technologies, free of…

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