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Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases.
Gene-editing therapies offer great hope for treating rare diseases, but they face big hurdles: the tremendous time and resources involved in devising a treatment that might only apply to a small number of patients.
A study published on Wednesday outlines a new approach that could make the process more efficient and less costly. Writing in the journal Nature, researchers presented a path toward a gene-editing strategy that could eventually be standardized for many different rare diseases, instead of personalized edits for each one.
“We are purposefully forgoing what is the most obvious way to treat a patient — fix their individual mutation back to the normal sequence,” said the study’s senior author, David R. Liu, a biologist at the Broad Institute and Harvard, who has pioneered several gene-editing advances.
Instead, he said, the idea is a “disease-agnostic” strategy: developing a technique that “could treat many more patients regardless of what mutation they have.”
There are more than 7,000 rare genetic diseases, conditions defined in the United States as affecting fewer than 200,000 people. Together, these diseases afflict about 30 million Americans and about 400 million people worldwide.
While the new approach is years away from potential use, it might ultimately apply to “a significant fraction” of those patients, conservatively about 10 percent, said Dr. Richard P. Lifton, the president of the Rockefeller University and head of its laboratory of human genetics and genomics.