Personalized Gene Editing to Treat an Inborn Error of Metabolism | NEJM
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.
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.
Audio Interview from the New England Journal of Medicine — Interview with Alice Chen on responding to the U.S. administration’s threats to health and physicians’…
Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated…
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.
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.
Throughout the course of multiple sclerosis, gradually progressive neurologic impairment can occur, which has been called disability accrual. Current disease-modifying therapies for multiple sclero…
(16 May, 2025) Free Nick Brown (12 March, 2025) Alison Clayton (19 January, 2025) Robert Wheeler (15 January, 2025) Free Charles Moorcroft, Abigail Whitehouse, Jonathan…
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Omar Noor, MD, FAAD and Randy Alevi, DO, discuss the identification and management of atopic dermatitis, with a focus on topical treatment options. They discuss the…
Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival…
Combined treatment with a tyrosine kinase inhibitor and blinatumomab has converted Ph-positive ALL, once the most deadly hematologic cancer, to a disease with long-term survival…