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The New England Journal of Medicine: Search Results in Genetics
The New England Journal of Medicine (NEJM) RSS feed -- Search Results in Genetics. NEJM ( is a weekly general medical journal that publishes new medical research findings, review articles, and editorial opinion on a wide variety of topics of importance to biomedical science and clinical practice.

Repurposing a Bacterial Immune System to Discover Antiviral Targets
31/12/69 - The effect of new, easy-to-use tools for editing cellular genomes in medicine and biomedical science is difficult to overstate. The new method uses programmable DNA-cutting machinery from a bacterial immune system (CRISPR [clustered regularly interspaced short palindromic repeats]?Cas9), which can…

Interleukin-12 and Interleukin-23 Blockade in Leukocyte Adhesion Deficiency Type 1
31/12/69 - LAD1 is a primary immunodeficiency resulting from mutations in ITGB2, which encodes the common CD18 subunit of the ?2 integrins; the ?2 integrins are required for the adhesion of neutrophils to endothelium and the transmigration of neutrophils into tissues. Patients with LAD1 typically have…

Breaking a Vicious Cycle
31/12/69 - In this issue of the Journal, Moutsopoulos et al. report on a patient with leukocyte adhesion deficiency type 1 (LAD1) who was successfully treated with ustekinumab, a monoclonal antibody to the p40 subunit common to interleukin-12 and interleukin-23. Patients with LAD1 have defective neutrophil…

Vision Loss after Intravitreal Injection of Autologous "Stem Cells" for AMD
31/12/69 - Age-related macular degeneration (AMD) is the leading cause of vision loss in persons older than 75 years of age in the United States. Progressive dysfunction and loss of retinal pigment epithelium cells and photoreceptors lead to poor visual acuity in patients with non-neovascular AMD. The…

Clarifying Stem-Cell Therapy?s Benefits and Risks
31/12/69 - The current excitement over the potential for stem-cell therapy to improve patient outcomes or even cure diseases is understandable. We at the Food and Drug Administration (FDA) share this excitement. However, to ensure that this emerging field fulfills its promise to patients, we must first…

Autologous Induced Stem-Cell?Derived Retinal Cells for Macular Degeneration
31/12/69 - Age-related macular degeneration (AMD) is one of the most prevalent retinal diseases that threaten vision in older populations in developed countries. Neovascular (also called "wet") AMD is more prevalent than atrophic (or "dry") AMD in Japan and is associated with the ectopic development of a…

Polar Extremes in the Clinical Use of Stem Cells
31/12/69 - The U.S. biotechnology and pharmaceutical industries arguably lead the world in innovation while operating under stringent regulations set by the Food and Drug Administration (FDA). Although flexible pathways exist to accelerate the development or approval of treatments for serious illnesses or…

Understanding Aging
31/12/69 - In 2006, Takahashi and Yamanaka reported the astonishing effect of forcing the simultaneous expression of a "cocktail" of four transcription factors (OCT4, SOX2, KLF4, and cMYC) on cells in the laboratory. This cocktail hijacked the controls and regulators that determine what a cell can or cannot…

Case 7-2017: A 73-Year-Old Man with Confusion and Recurrent Epistaxis
31/12/69 - Presentation of Case. Dr. Jeffrey L. Greenwald: A 73-year-old man with multiple chronic medical illnesses was admitted to this hospital because of confusion and irritability. Two days before this admission, increasing weakness, lethargy, chills, and diarrhea developed. The patient became less…

Gene Therapy in a Patient with Sickle Cell Disease
31/12/69 - Sickle cell disease is among the most prevalent inherited monogenic disorders. Approximately 90,000 people in the United States have sickle cell disease, and worldwide more than 275,000 infants are born with the disease annually. Sickle cell disease was the first disease for which the molecular…

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