Work begins on boosting adoption of gene editing technologies

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A new program at the National Human Genome Research Institute within the National Institutes of Health hopes to increase adoption of genome editing technologies for treating disease, with up to $190 million available to researchers over six years.

Genome editing is a molecular approach to make precise changes to the sequence of DNA, which is the hereditary material inside live cells. Editing can change how DNA interacts with RNA, which carries instructions from DNA to control proteins.

The new sequence can change the course of a disease in a person who inherited a disease. For example, a particular sequence in a patient with brain degeneration could slow the degeneration over time.

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These technologies hold great promise, including the possibility of editing the disease-causing DNA within the non-reproductive “somatic” cells of the body,” according to NIH literature. “Such changes would not be passed down to future generations.”

The Somatic Cell Genome Editing program just launched has four core missions:

• Expand the number of genome editing tools available to researchers.

• Develop delivery systems that can target the cells of specific organs and tissues in the human body efficiently.

• Design new assays for testing safety and efficacy of editing and delivery tools.

• Distribute the knowledge, methods and tools that have been developed through this program to the scientific community.

“Think of machines going into the cell and editing it, and regulating activity,” explains P.J. Brooks, the program coordinator at NIH. “The challenge now is finding a delivery system to put the changed cell back in the organ from where it came while avoiding other cells, including reproductive cells.”

The long-term goal is to develop field trials then bring the technology into physician offices for testing with the hope that gene editing can become common practice.

So far, research of medical literature on delivering the cell tissue back in the body has been difficult, says Mary Ellen Perry, a program director at NIH. “Right now, there is no way to search online—the data is disbursed and not commonly agreed to. The literature is scattered all over.”

Consequently, the project now will focus on development of a centralized online toolbox—a web site—to store results of research and developments learned in-house and from other projects. “We want to find researchers who know how to make a toolkit, and we will support them in building it,” Perry says.

The project on January 24 issued a call for researchers and other scientists to submit their projects and ideas, with a due date of April 3. An informational web seminar via WebEx is scheduled on February 16. The call-in number is 623-436-972 and the password is SCGE2018.

The NIH program will consider the proposals with funding decisions expected in September and an anticipation of 25 to 35 funding awards being made. “We want investigators to collaborate on the web site,” Brooks says.

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