DENVER - National Jewish Health researchers with the Advanced Diagnostic Laboratories have developed a groundbreaking molecular diagnostic test capable of accurately diagnosing a major genetic cause ...

Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency—a promising leap toward treating humans with this rare ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Considering a risk score generated from multiple genetic variants linked to chronic obstructive pulmonary disease (COPD) caught undiagnosed cases of the disease better than conventional risk factors ...

α1-Antitrypsin deficiency is a hereditary disorder that affects the liver in children and adults, as well as the lungs in adults. The disease is mostly caused by the Z allele mutation in the SERPINA1 ...

Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by ...

Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency - a promising leap toward treating humans with this rare ...