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41. 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.
42. 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).
44. 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.
46. IVUS-Guided versus Angiography-Guided PCI in Unprotected Left Main Coronary Disease.
作者: Luca Testa.;Jose Maria De la Torre Hernandez.;Giovanni Luigi De Maria.;Daniel A Jones.;Pablo Piñon-Esteban.;Gianluca Campo.;Bruno Garcia Del Blanco.;Manuel Pan.;Tamara Garcia-Camarero.;Gennaro Sardella.;Peter O'Kane.;John P Greenwood.;Flavio L Ribichini.;Irene Pescetelli.;Alfonso Ielasi.;Iñigo Lozano.;James Cockburn.;Jacopo A Oreglia.;Azfar G Zaman.;Francesco Bedogni.;Wietze Lindeboom.;Jan G P Tijssen.;Ernest Spitzer.;Adrian P Banning.; .
来源: N Engl J Med. 2026年
Percutaneous coronary intervention (PCI) is increasingly used for revascularization of unprotected left main coronary artery disease. Whether intravascular ultrasonographic (IVUS) guidance during PCI results in better clinical outcomes than conventional angiographic guidance alone is uncertain.
47. Intravascular Ultrasound-Guided or Angiography-Guided Complex High-Risk PCI.
作者: Roberto Diletti.;Joost Daemen.;Benjamin Faurie.;Marco Barbierato.;Didier Tchétché.;Thomas Hovasse.;Koen Teeuwen.;Jin M Cheng.;Martin Landt.;Gianluca Campo.;Johan Bennett.;Fernando Alfonso.;Kambis Mashayekhi.;Raul Moreno.;Youssef S Abdelwahed.;Ton Heestermans.;Jose M de la Torre Hernandez.;John C Murphy.;Ignacio Amat-Santos.;Joseph Dens.;Alfonso Franzé.;David M Leistner.;Angie Ghattas.;James C Spratt.;Adrian P Banning.;Jan G P Tijssen.;Ernest Spitzer.;Nicolas M Van Mieghem.; .
来源: N Engl J Med. 2026年
Intravascular ultrasound (IVUS) guidance during percutaneous coronary intervention (PCI) has been associated with increased stent optimization and reduced adverse events among patients with complex coronary-artery lesions, but adoption of this strategy in Western countries remains low. Although practice guidelines recommend intracoronary imaging for anatomically complex lesions, evidence from current European practice is limited.
50. Discontinuation of Beta-Blocker Therapy after Myocardial Infarction.
作者: Ki Hong Choi.;Danbee Kang.;Weon Kim.;Joon-Hyung Doh.;Juhan Kim.;Yong Hwan Park.;Sung Gyun Ahn.;Jong Pil Park.;Sang Min Kim.;Byung Ryul Cho.;Chang-Wook Nam.;Jang Hyun Cho.;Seung-Jae Joo.;Jon Suh.;Jin-Ok Jeong.;Woo Jin Jang.;Choong Won Goh.;Chang-Hwan Yoon.;Jin-Yong Hwang.;Seong-Hoon Lim.;Sang Rok Lee.;Eun-Seok Shin.;Byung Jin Kim.;Cheol Woong Yu.;Sung-Ho Her.;Hyun Kuk Kim.;Kyu Tae Park.;Juwon Kim.;Jihoon Kim.;Taek Kyu Park.;Joo Myung Lee.;Juhee Cho.;Jeong Hoon Yang.;Young Bin Song.;Seung-Hyuk Choi.;Hyeon-Cheol Gwon.;Eliseo Guallar.;Joo-Yong Hahn.; .
来源: N Engl J Med. 2026年394卷13期1302-1312页
The role of long-term beta-blocker therapy after a myocardial infarction in patients without left ventricular systolic dysfunction or heart failure is unclear in the era of contemporary coronary-artery reperfusion and secondary prevention interventions.
51. Iptacopan in IgA Nephropathy - Final 24-Month Data.
作者: Jonathan Barratt.;Necmi Eren.;Naoki Kashihara.;Bart Maes.;Dana V Rizk.;Brad Rovin.;Hernán Trimarchi.;Hong Zhang.;Weiming Wang.;Ismail Kocyigit.;Chuanming Hao.;Vladimir Tesař.;Kenan Turgutalp.;Li Yang.;Guangqun Xing.;Valter Duro Garcia.;Seung Hyeok Han.;Wanhong Lu.;Antonio Pisani.;Julia Weinmann-Menke.;Frank Eitner.;Nicolas Guerard.;Dmytro Butylin.;Luca Monaco.;Emil Scosyrev.;Annabel Magirr.;Ronny Renfurm.;Thomas Hach.;Vlado Perkovic.; .
来源: N Engl J Med. 2026年
Overactivation of the alternative complement pathway contributes to IgA nephropathy and glomerular inflammation. In the 9-month interim analysis of this phase 3 trial, iptacopan, a complement factor B inhibitor, led to a significant reduction of 38.3% in the 24-hour urinary protein-to-creatinine ratio as compared with placebo and had an acceptable safety profile.
52. Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy.
作者: Joseph W Rossano.;Charles Canter.;Cordula M Wolf.;Andrew Papez.;Marta Gambra.;Randall M Bryant.;Juan Alejos.;Michael McCulloch.;Georgia Sarquella Brugada.;Matthew J Bock.;Aamir Jeewa.;F Bennett Pearce.;Milind Y Desai.;Nicholas Favatella.;Atefeh Javidialsaadi.;Van Phung.;Thomas Rano.;Lin Zhu.;Joshua L Dyme.;Seema Mital.; .
来源: N Engl J Med. 2026年
Approved pharmacologic therapies for pediatric hypertrophic cardiomyopathy are lacking, and surgical intervention may be indicated in patients with left ventricular outflow tract obstruction. The efficacy and safety of mavacamten, a cardiac myosin inhibitor that is available for adults, warrant evaluation in adolescents.
55. Angiography-Derived Fractional Flow Reserve to Guide PCI.
作者: William F Fearon.;Allen Jeremias.;Guy Witberg.;Rasha Al-Lamee.;David J Cohen.;Amir Kaki.;Rahul P Sharma.;Robert W Yeh.;Bassem M Chehab.;Michael C Kim.;Hiromasa Otake.;Rajiv Tayal.;Hitoshi Matsuo.;Margaret McEntegart.;Ankitkumar K Patel.;Yader Sandoval.;Karim M Al-Azizi.;Kazuhiro Dan.;Louai Razzouk.;Stephane Fournier.;Alexandra Popma.;Jeffrey J Popma.;Duzhi Zhao.;Bjorn Redfors.;Mahesh V Madhavan.;Ran Kornowski.;Martin B Leon.;Ajay J Kirtane.; .
来源: N Engl J Med. 2026年
Assessing intermediate coronary lesions with an intracoronary pressure wire improves clinical outcomes in patients undergoing cardiac catheterization and percutaneous coronary intervention (PCI). However, clinical use of pressure-wire-based physiological assessment remains low. Measurement of fractional flow reserve (FFR) derived from coronary angiographic images alone correlates well with pressure-wire-based FFR measurements and may simplify procedures, but its effect on clinical outcomes is unknown.
56. Angiography-Based Physiology to Guide Coronary Revascularization.
作者: Joost Daemen.;Jari A van der Eijk.;Marco Barbierato.;Robert A Byrne.;Paolo Canova.;Giovanni L De Maria.;Eric Van Belle.;Ignacio Amat-Santos.;Antonio Mangieri.;Thomas Hovasse.;Manel Sabaté.;Ronan Margey.;Simone Biscaglia.;Fernando Rivero.;Domenico D'Amario.;Youssef S Abdelwahed.;Rohit M Oemrawsingh.;Raul Moreno.;James Cockburn.;Luca Testa.;Ton de Vries.;Ulf Landmesser.;Adrian P Banning.;Jan G P Tijssen.;Ernest Spitzer.; .
来源: N Engl J Med. 2026年
Current society guidelines recommend physiological assessment of intermediate coronary lesions to guide revascularization. Data regarding revascularization guided by vessel fractional flow reserve (vFFR), derived from three-dimensional quantitative coronary angiography without the need for a pressure wire or hyperemic agent, as compared with pressure-wire-based fractional flow reserve (FFR), are lacking.
57. Left Ventricular Unloading in High-Risk Percutaneous Coronary Intervention.
作者: Divaka Perera.;Matthew Ryan.;Saad M Ezad.;Sohail Q Khan.;Ian Webb.;Peter D O'Kane.;Roshan Weerackody.;Matthew Dodd.;Matthew Kwok.;Lynn Laidlaw.;Laura Van Dyck.;Benjamin Wrigley.;Julian W Strange.;Alan Bagnall.;Farzin Fath-Ordoubadi.;Vasileios F Panoulas.;Andrew Ladwiniec.;John R Davies.;Alexander Chase.;Colum G Owens.;Stuart Watkins.;Haseeb Rahman.;Nilesh Pareek.;Krishnaraj Rathod.;John Rawlins.;Richard Evans.;Stephen P Hoole.;Rod H Stables.;Nick Curzen.;Tim Clayton.; .
来源: N Engl J Med. 2026年
Complex percutaneous coronary intervention (PCI) in patients with severely impaired left ventricular function carries a high risk of death and complications. Whether percutaneous left ventricular unloading improves outcomes remains unclear.
59. Left Atrial Appendage Closure or Anticoagulation for Atrial Fibrillation.
作者: Shephal K Doshi.;Saibal Kar.;Devi G Nair.;Thomas Waggoner.;Himanshu Agarwal.;Mehran Moussavian.;Amir Kashani.;Saumil Oza.;Leon Feldman.;Ashish Sadhu.;David DeLurgio.;Oluseun Alli.;Jens Erik Nielsen-Kudsk.;Masanori Yamamoto.;Mohamad Alkhouli.;A John Camm.;Megan Coylewright.;C Michael Gibson.;Christopher B Granger.;Mahmut Edip Gurol.;Kenneth C Huber.;Moussa Mansour.;Andrea Natale.;Stuart J Pocock.;Vivek Y Reddy.;Walid I Saliba.;Federico M Asch.;Scott Wehrenberg.;Kate Frost.;Thomas Christen.;Brad S Sutton.;Kenneth M Stein.;Martin B Leon.;Kenneth A Ellenbogen.; .
来源: N Engl J Med. 2026年
For patients with atrial fibrillation, the use of oral anticoagulant therapy to prevent stroke is limited by the risk of bleeding. Left atrial appendage closure is considered for patients who are unsuitable candidates for long-term anticoagulation, but its role in patients who are eligible for anticoagulants has not been established.
60. Ultrasound-Facilitated, Catheter-Directed Fibrinolysis for Acute Pulmonary Embolism.
作者: Kenneth Rosenfield.;Frederikus A Klok.;Gregory Piazza.;Andrew S P Sharp.;Fionnuala Ní Áinle.;Michael R Jaff.;Stefano Barco.;Samuel Z Goldhaber.;Nils Kucher.;Irene M Lang.;Irene Schmidtmann.;Keith M Sterling.;Aleksander Araszkiewicz.;Vishal Arora.;Rafael Cires-Drouet.;John Coghlan.;Lukas Hobohm.;Wulf D Ito.;Kurt Jacobson.;Christoph Kaiser.;Grzegorz Kopec.;Kristin Marx.;Samuel McElwee.;Nicolas Meneveau.;Peter Monteleone.;Jose M Montero-Cabezas.;Christoph B Olivier.;John Park.;Marek Roik.;Rahul Sakhuja.;Andi Tego.;Markus Theurl.;Gautam Visveswaran.;Jan Albert Vos.;Michael N Young.;Federico M Asch.;Stavros V Konstantinides.; .
来源: N Engl J Med. 2026年
Whether anticoagulation alone is an adequate treatment for acute, intermediate-risk pulmonary embolism is uncertain.
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