In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder. 2 Department of Chemistry and Biochemistry, University of ...

Tirzepatide and semaglutide are highly effective medications for obesity management. The efficacy and safety of tirzepatide as compared with semaglutide in adults with obesity but without type 2 ...

Hypoxemia occurs early during an attack and, as indicated by the low carbon dioxide tensions, is not the result of overall underventilation. In that regard asthmatic patients act like the group of ...

Christopher Duggan is the director of the Center for Nutrition at Boston Children’s Hospital, a professor of pediatrics at Harvard Medical School, and a professor in the Departments of Nutrition ...

Chimeric antigen receptor (CAR) T cells targeting CD19 have transformed the treatment of B-cell cancers, but many patients do not have long-term remission. We designed an anti-CD19 enhanced ...

Dr. Maxwell Green (Medicine): A 70-year-old man presented to the emergency department of this hospital because of a 6-week history of anorexia, weakness, and weight loss. Six weeks before this ...

These epidemiologic patterns highlight the limitations of focusing on genetics alone. Although the Human Genome Project greatly expanded our understanding of hereditary contributions to disease ...

Obicetrapib is a highly selective cholesteryl ester transfer protein inhibitor that reduces low-density lipoprotein (LDL) cholesterol levels. The efficacy and safety of obicetrapib have not been ...

Mycobacterium tuberculosis continues to cause substantial illness globally. The efficacy of BCG revaccination for the prevention of sustained M. tuberculosis infection is unclear. New research ...

Medical House Pupil, Massachusetts General Hospital, 1901; Assistant in Pediatrics, Harvard University; Assistant Physician at the Children's Hospital; Assistant Physician and Pathologist at the ...

Lepodisiran is a long-duration small interfering RNA that inhibits hepatic production of apolipoprotein(a). New research findings on lepodisiran in patients with elevated lipoprotein(a ...

Arthur Robin Williams is an associate professor of clinical psychiatry at Columbia University and a research scientist at the New York State Psychiatric Institute. Stephen Morrissey, the ...