New research results show that a new form of gene editing is efficient in correcting a mutation in patient cells with the monogenic disease Alpha-1 antitrypsin deficiency (AATD), a common inherited disease that affects the liver and the lungs. This new approach, called base editing, is different from other forms of editing including CRISPR, because the base editors do not induce a break in the DNA, which helps prevent double strand breaks, potential off-target editing, and unwanted mutations during cell repair.
Led by Andrew Wilson, MD and Rhiannon Werder, Ph.D. of the Center for Regenerative Medicine (CReM) at Boston Medical Center and Boston University, the researchers utilized patient-derived liver cells termed “iHeps”, that mimic the biology of liver hepatocytes, the main producers of alpha-1 antitrypsin protein in the body that likewise perform important metabolic, endocrine and secretory functions. The base editing technology corrected the Z mutation responsible for AATD and reduced the effects of the disease in the hepatocytes, demonstrating successful base editing in human cells. These results, published in Molecular Therapy, can help pave the way for future human trials.
“This study shows the successful application of base editing technology to correct the mutation responsible for AATD in liver cells derived from patients with this disease,” says Wilson, a pulmonologist at Boston Medical Center and an associate professor of medicine at Boston University School of Medicine, who served as the study’s corresponding author. “I am hopeful that these results will create a pathway to use this technology to help patients with AATD and other monogenic diseases.”
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