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STAT+: Beam Therapeutics says CRISPR treatment shows encouraging results in lung condition
Beam Therapeutics said it used a form of CRISPR called base editing to correct, in several patients, a mutation that drives a debilitating lung condition.
Beam Therapeutics said Monday that it used a form of CRISPR called base editing to correct, in several patients, a mutation that drives a debilitating lung condition that may affect tens of thousands of Americans.
The company touted the data as evidence of the first successful use of CRISPR gene editing to fix a disease-causing mutation. Most previous CRISPR trials have focused on knocking out genes that are either deleterious or that, when removed, can have profound impacts in particular diseases, like sickle cell.
Patients with the condition Bean targeted, alpha-1 antitrypsin deficiency, or AATD, generally have a single misspelled letter in the gene that codes for AAT, a protein that helps protect the lungs.
