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Photo: Co-first author Dewan Shrestha (left), corresponding author Dr.
Image source: Courtesy of St.
Scientists at St.
"We've created Google Maps that edit genomes," said co-corresponding author Dr.
Gene therapy, which is giving a patient a normal copy of a dysfunctional gene, has proven successful
in treating certain genetic disorders.
A new way to find a safe port location
The tool uses data from the 1000 Genomes Project to compare highly variable DNA sequences
between healthy people.
Cheng said: "Our method is a new way to identify genomic safe harbor sites in an organization-specific way
.
If the DNA in a single cell is a rope, it will be two meters long
.
"Our tool evaluates the three-dimensional structure of DNA because human DNA is not a one-dimensional linear structure, it is actually three-dimensional," Chen said
.
Balance safety and therapeutic gene expression
"Safe gene therapy requires two things, first, to maintain high expression
of new genes.
The scientists found that these genes were placed in safe harbor locations identified by their tools, and their expression remained unchanged
over time.
The tool, known as the Genomics and Epigenetics Guided Safe Harbor Cartographer (GEG-SH Cartographer), is available
free of charge in https://github.
The study's first authors are Dewan Shrestha of the Health Sciences Center at the University of St.
essay
Genomics and epigenetics guided identification of tissue-specific genomic safe harbors
