jueves, 18 de abril de 2019

There could be a middle route in the genome editing debate

The Readout
Damian Garde

There could be a middle route in the genome editing debate


The world of science has long since accepted the idea of using CRISPR to edit the genomes of adults, but tinkering with the DNA of embryos is the subject of a global moratorium. There might be a way to split the difference: using CRISPR at the fetal stage to make edits that won’t be passed down through generations.

As STAT’s Sharon Begley reports, scientists at Children’s Hospital of Philadelphia injected CRISPR into the amniotic fluid of pregnant mice in hopes of editing a genetic defect that leads to lung disease. It worked in about 20% of them, which appears unimpressive on its face, but the researchers see it as a promising proof of concept that will lay a path for more experiments.

The idea is years away from being tested in humans, let alone approved for use, but it highlights CRISPR’s potential to treat diseases so severe that even the first months of life are too late for intervention.

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