9. Digital Twin-Guided Ablation for Ventricular Tachycardia.
作者: Jonathan Chrispin.;Adityo Prakosa.;Eugene Kholmovski.;Aravindan Kolandaivelu.;Amanda Barcelon.;Konstantinos N Aronis.;Ronald D Berger.;Hugh Calkins.;Natalia Trayanova.
来源: N Engl J Med. 2026年394卷13期1345-1347页 15. GLP-1 Receptor Agonists.
Glucagon-like peptide-1 (GLP-1) receptor agonists are incretin analogues that promote glucose-mediated insulin release and are used to treat type 2 diabetes mellitus and obesity. GLP-1 receptor agonists and GLP-1 and glucose-dependent insulinotropic peptide agonists have several mechanisms of action, including reduction of gastric emptying, inhibition of glucagon secretion, beneficial changes in the intestinal microbiome, and direct effects on hypothalamic nuclei to enhance satiety (which promotes weight loss). Beyond the impressive effects of GLP-1 receptor agonists on blood glucose levels and body weight, large-scale randomized, controlled trials have shown that GLP-1 receptor agonists reduce cardiovascular risk and slow progression to renal failure in persons at high risk and those with type 2 diabetes. Adverse side effects from GLP-1 receptor agonists are mostly gastrointestinal but may also include loss of muscle and bone mass. Questions remain about long-term adherence, weight regain after discontinuation of treatment, and the functional implications of the loss of muscle and bone mass. Recent and ongoing targeted studies suggest the possibility of additional uses for GLP-1 receptor agonists.
16. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia.
作者: Haydar Frangoul.;Rabi Hanna.;Mark C Walters.;Roy L Kao.;Clinton Carroll.;Meghann McManus.;Kai-Hsin Chang.;Michael C Jaskolka.;Keunpyo Kim.;Qifeng Yu.;Nnenna Badamosi.;Baisong Mei.;Olubunmi Afonja.;Alexis Thompson.; .
来源: N Engl J Med. 2026年394卷13期1292-1301页
Renizgamglogene autogedtemcel (reni-cel) is an investigational clustered regularly interspaced short palindromic repeats (CRISPR)-Cas12a gene-edited autologous hematopoietic stem-cell therapy. The therapy was designed to disrupt the BCL11A binding sites in the HBG1 and HBG2 promoters to reactivate fetal hemoglobin production for the treatment of transfusion-dependent β-thalassemia.
17. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.
作者: Rabi Hanna.;Haydar Frangoul.;Luis Pineiro.;Christopher McKinney.;Markus Mapara.;Jignesh Dalal.;Hemalatha G Rangarajan.;Harold Atkins.;Akshay Sharma.;Kai-Hsin Chang.;Michael C Jaskolka.;Keunpyo Kim.;Qifeng Yu.;Baisong Mei.;Olubunmi Afonja.;Mark C Walters.; .
来源: N Engl J Med. 2026年394卷13期1281-1291页
Renizgamglogene autogedtemcel (reni-cel) is an investigational clustered regularly interspaced short palindromic repeats (CRISPR)-Cas12a gene-edited autologous hematopoietic stem-cell therapy. The therapy was designed to disrupt the BCL11A binding sites in the HBG1 and HBG2 promoters to reactivate fetal hemoglobin production for the treatment of sickle cell disease.
18. Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease.
作者: Ashish O Gupta.;Akshay Sharma.;Haydar Frangoul.;Julie Kanter.;Markus Y Mapara.;Jignesh Dalal.;Asif Alavi.;Jennifer J Jaroscak.;Ernesto Ayala.;John F DiPersio.;Edward D Ziga.;Mary Eapen.;Stacey Rifkin-Zenenberg.;Alex C Minella.;Yinzhong Chen.;Sarah Chesler.;Srikanth Ambati.;Thomas S Bowman.;Bahru Habtemariam.;Marcelyne Joseney-Antoine.;Priya S Chockalingam.;Ling Lin.;Sunita Goyal.;Amy Simon.;Alexis A Thompson.;Matthew M Heeney.; .
来源: N Engl J Med. 2026年
Sickle cell disease is characterized by chronic hemolytic anemia and recurrent severe vaso-occlusive crises. Ristoglogene autogetemcel (risto-cel) includes autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited to target the HBG1 and HBG2 promoters and inhibit BCL11A binding without altering BCL11A expression, yielding a switch in hemoglobin production from sickle hemoglobin (HbS) to antisickling fetal hemoglobin (HbF).
20. Sirolimus-Coated Balloon Angioplasty for Infrainguinal Artery Disease.
作者: Stefano Barco.;Rolf P Engelberger.;Ulrike Held.;Riccardo M Fumagalli.;Alexandru Grigorean.;Daniel Hayoz.;Mario Münger.;Tim Sebastian.;Roman Stadler.;Davide Voci.;Simon Wolf.;Frédéric Baumann.;Tobias Tritschler.;Conrad von Stempel.;Daniel Périard.;Nils Kucher.; .
来源: N Engl J Med. 2026年
Whether sirolimus-coated balloon angioplasty for infrainguinal artery disease reduces the incidence of major adverse limb events remains unknown.
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