Alnylam Pharmaceuticals this week presented preclinical data on its investigational treatment for alpha-1 antitrypsin deficiency-associated liver disorders.

AAT is a liver-produced serine proteinase inhibitor that protects the lungs from neutrophil elastase and other irritants that cause inflammation, according to Alnylam. In the liver, misfolding of the mutant form of the protein disrupts its normal release into the blood, causing it to aggregate in hepatocytes. The result can be liver injury, fibrosis, cirrhosis, and liver cancer.

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The US Food and Drug Administration has approved Kite Pharmaceuticals' CAR T-cell therapy for large B-cell lymphomas, the New York Times reports.

Kaiser Health News reports that gene therapies could cost more than a million dollars.

Worcester Polytechnic Institute researchers have received a grant to combine biology and computer science for high school students.

In Nature this week: variants associated with obsessive-compulsive disorder, review of key CRISPR enzymes, and more.