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1. 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.
2. 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.
3. 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.
4. Early Surgery or Conservative Care for Asymptomatic Aortic Stenosis at 10 Years.
作者: Duk-Hyun Kang.;Sung-Ji Park.;Ga Yun Kim.;Sahmin Lee.;Byung Joo Sun.;Joon Bum Kim.;Sung-Ho Jung.;Hyung-Kwan Kim.;Sung-Cheol Yun.;Geu-Ru Hong.;Jong-Min Song.;Cheol-Hyun Chung.
来源: N Engl J Med. 2026年394卷12期1167-1174页
Among asymptomatic patients with severe aortic stenosis, a previous analysis showed that the risk of a composite of death during surgery or within 30 days after surgery (called operative mortality) or death from cardiovascular causes was significantly lower with early surgery than with conservative care. However, the long-term survival benefit of early surgery, as compared with conservative care, remains unclear.
5. Atezolizumab plus FOLFOX for Stage III Mismatch Repair-Deficient Colon Cancer.
作者: Frank A Sinicrope.;Fang-Shu Ou.;Dirk Arnold.;Walter R Peters.;Robert J Behrens.;Christopher H Lieu.;Khalid Matin.;Deirdre J Cohen.;Samara L Potter.;Andrew B Nixon.;Lisa A Kottschade.;Emily Kathol.;Wendy L Frankel.;Ardaman Shergill.;Dennis Hsu.;Anke Reinacher-Schick.;Paul Mehan.;Philip J Gold.;Maged F Khalil.;Tyler Zemla.;Clare Gatten.;Eileen M O'Reilly.;Jeffrey A Meyerhardt.
来源: N Engl J Med. 2026年394卷12期1155-1166页
Standard adjuvant chemotherapy for stage III colon cancer consists of a fluoropyrimidine-plus-oxaliplatin regimen. Whether the addition of atezolizumab (an anti-programmed death ligand 1 agent) to a modified FOLFOX6 regimen (fluorouracil, oxaliplatin, and leucovorin; called mFOLFOX6) would improve outcomes in patients with stage III colon cancer with mismatch repair-deficient (dMMR) status is unclear.
6. A Placebo-Controlled Trial of the Oral PCSK9 Inhibitor Enlicitide.
作者: Ann Marie Navar.;Elina Mikhailova.;Alberico L Catapano.;Puja Banka.;Dirk J Blom.;Alberto Cadena.;Susan Kourpanidis.;Norman E Lepor.;Kazuhisa Tsukamoto.;Geraldine Mendizabal.;Julio Nunez.;Wenjuan Zhang.;Pengfei Zhu.;Min Zhuo.;Christie M Ballantyne.; .
来源: N Engl J Med. 2026年394卷6期529-539页
Enlicitide decanoate, an oral proprotein convertase subtilisin-kexin type 9 (PCSK9) inhibitor, was shown to reduce low-density lipoprotein (LDL) cholesterol levels in a phase 2 trial; longer-term data are needed.
7. Left Atrial Appendage Closure or Medical Therapy in Atrial Fibrillation.
作者: Ulf Landmesser.;Carsten Skurk.;Paulus Kirchhof.;Thorsten Lewalter.;Johannes Hartung.;Andi Rroku.;Burkert Pieske.;Johannes Brachmann.;Ibrahim Akin.;Claudius Jacobshagen.;Benjamin Meder.;Andreas Zeiher.;Stefan D Anker.;Holger Thiele.;Stefan Blankenberg.;Steffen Massberg.;Heribert Schunkert.;Norbert Frey.;Alexander Joost.;Martin Bergmann.;Ralph Stephan von Bardeleben.;Tim Friede.;Marius Placzek.;Anna Suling.;Karl Georg Haeusler.;Matthias Endres.;Karl Wegscheider.;Leif-Hendrik Boldt.;Ingo Eitel.; .
来源: N Engl J Med. 2026年394卷13期1270-1280页
Catheter-based closure of the left atrial appendage is an alternative to oral anticoagulation for stroke prevention in patients with atrial fibrillation. The effectiveness of this strategy, as compared with physician-directed best medical care, in patients at high risk for stroke and bleeding is unknown.
8. Romiplostim versus Placebo for Chemotherapy-Induced Thrombocytopenia.
作者: Hanny Al-Samkari.;César Muñoz.;Çağlayan Geredeli.;Ippokratis Korantzis.;Beatriz González Astorga.;Cagatay Arslan.;Johnny Francisco Cordeiro Camargo.;Florian Scotté.;Giuliano Borges.;Kejia Wang.;Melissa Eisen.;David J Kuter.;Gerald A Soff.
来源: N Engl J Med. 2026年394卷11期1061-1073页
Chemotherapy-induced thrombocytopenia (CIT) is a common complication of chemotherapy that is associated with bleeding, reduced relative dose intensity, and potentially worse outcomes. No widely available therapies are approved for CIT.
9. Bleeding Risk with Apixaban vs. Rivaroxaban in Acute Venous Thromboembolism.
作者: Lana A Castellucci.;Vivien M Chen.;Michael J Kovacs.;Alejandro Lazo-Langner.;Peter Greenstreet.;Susan Kahn.;Benoit Côté.;Sam Schulman.;Kerstin de Wit.;James Douketis.;Deepa Suryanarayan.;Tony Wan.;Erik Yeo.;Genevieve Le Templier.;Huyen A Tran.;Abbey Willcox.;Helen J Crowther.;Ritam Prasad.;Sudeep Shivakumar.;Etimbuk Umana.;Fionnuala Ni Ainle.;Tobias Tritschler.;Stefano Barco.;Jean-Philippe Galanaud.;Marc Blondon.;Lisa Baumann Kreuziger.;Susan Solymoss.;Clive Kearon.;Erin Thomas.;Tim Ramsay.;Gregoire Le Gal.;Marc Rodger.; .
来源: N Engl J Med. 2026年394卷11期1051-1060页
Apixaban and rivaroxaban are the oral anticoagulants most frequently used to treat acute venous thromboembolism. However, uncertainty remains about the difference in bleeding risk between the two medications.
10. Zorevunersen in Children and Adolescents with Dravet Syndrome.
作者: Linda Laux.;Joseph Sullivan.;M Scott Perry.;Andreas Brunklaus.;Archana Desurkar.;John M Schreiber.;Colin M Roberts.;Kelly G Knupp.;James W Wheless.;Elaine C Wirrell.;Pam Ventola.;Fei Wang.; Meena.;Jessie Lynch.;Kimberly A Parkerson.;Barry Ticho.;J Helen Cross.; .
来源: N Engl J Med. 2026年394卷10期969-982页
Dravet syndrome is a severe developmental and epileptic encephalopathy caused primarily by SCN1A haploinsufficiency. Risks of sudden unexpected death in epilepsy and cognitive deficits are higher among patients with this syndrome than in the general population with epilepsy. The effects of zorevunersen, an antisense oligonucleotide designed to up-regulate NaV1.1 sodium channels, in patients with Dravet syndrome are not known.
11. Neoadjuvant GOLP in Resectable High-Risk Intrahepatic Cholangiocarcinoma.
作者: Guo-Ming Shi.;Xiao-Yong Huang.;Fei Liang.;Xiao Liang.;Rui Dong.;Qing-Hai Ye.;Qiang Gao.;Yuan Ji.;Zheng-Ping Yu.;Wen-Long Zhai.;Jia-Cheng Lu.;Xiao-Wu Li.;Fu-Bao Liu.;Kui Wang.;Wei Yang.;Jia-Lin Zhang.;Zhi-Qiang Hu.;Shuang-Jian Qiu.;Xiao-Ying Wang.;Xin-Rong Yang.;Hui-Chuan Sun.;Ying-Hong Shi.;Zhen-Bin Ding.;Wei-Ren Liu.;Xiao-Wu Huang.;Cheng Huang.;Ying-Hao Shen.;Jian Sun.;Yi-Feng He.;Yuan-Fei Peng.;Yang Xu.;Jian-Jun Zou.;Jian Zhou.;Jia Fan.; .
来源: N Engl J Med. 2026年394卷10期983-995页
No neoadjuvant treatment has been considered to be standard therapy for patients with resectable intrahepatic cholangiocarcinoma with high-risk factors for recurrence. The GOLP regimen (gemcitabine-oxaliplatin, lenvatinib, and an anti-programmed death 1 antibody) has shown promising efficacy with a manageable safety profile in advanced intrahepatic cholangiocarcinoma and biliary tract cancer.
12. Finerenone in Type 1 Diabetes and Chronic Kidney Disease.
作者: Hiddo J L Heerspink.;Andreas L Birkenfeld.;David Z I Cherney.;Helen M Colhoun.;Per-Henrik Groop.;Linong Ji.;Niels Jongs.;Chantal Mathieu.;Richard E Pratley.;Sylvia E Rosas.;Peter Rossing.;Jay S Skyler.;Katherine R Tuttle.;Robert Lawatscheck.;Meike Brinker.;Markus F Scheerer.;Julie Russell.;Patrick Schloemer.;Janet B McGill.; .
来源: N Engl J Med. 2026年394卷10期947-957页
The nonsteroidal mineralocorticoid receptor antagonist finerenone has been reported to improve kidney and cardiovascular outcomes in persons with type 2 diabetes and chronic kidney disease (CKD). The efficacy and safety of finerenone in persons with type 1 diabetes and CKD are unknown.
13. Phase 1 Study of Rezatapopt, a p53 Reactivator, in TP53 Y220C-Mutated Tumors.
作者: Ecaterina E Dumbrava.;Geoffrey I Shapiro.;Aparna R Parikh.;Melissa L Johnson.;Anthony W Tolcher.;John A Thompson.;Anthony B El-Khoueiry.;Andrae L Vandross.;Shivaani Kummar.;Dale R Shepard.;Kim LeDuke.;Lisa Sheehan.;Leila Alland.;Arshad Haque.;Deepika Jalota.;Marc Fellous.;Alison M Schram.
来源: N Engl J Med. 2026年394卷9期872-883页
Rezatapopt is an investigational, first-in-class, oral, selective p53 reactivator that specifically binds to Y220C-mutated p53, which stabilizes p53 in its wild-type conformation and restores its functionality.
14. Perioperative Enfortumab Vedotin and Pembrolizumab in Bladder Cancer.
作者: Christof Vulsteke.;Nabil Adra.;Pongwut Danchaivijitr.;Maksym Sabadash.;Alejo Rodriguez-Vida.;Zhentao Zhang.;Vagif Atduev.;Y Emre Göger.;Steffen Rausch.;Seok-Ho Kang.;Yohann Loriot.;Jens Bedke.;Matthew D Galsky.;Peter H O'Donnell.;Gunhild von Amsberg.;Nimira Alimohamed.;Grzegorz Sulimka.;Shilpa Gupta.;Viktor Paramonov.;Keita Nakane.;Michael Mihm.;Changting Meng.;Caizhi David Huang.;Chethan Ramamurthy.;Blanca Homet Moreno.;Anders Ullén.; .
来源: N Engl J Med. 2026年394卷13期1257-1269页
Patients with muscle-invasive bladder cancer who are ineligible for cisplatin-based chemotherapy proceed directly to radical cystectomy with pelvic lymph-node dissection. Perioperative therapy may improve outcomes in this population.
15. An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1.
作者: Nicholas E Johnson.;Li-Jung Tai.;Johanna I Hamel.;John W Day.;Jeffrey M Statland.;Payam Soltanzadeh.;Sankarasubramoney H Subramony.;Charles A Thornton.;W David Arnold.;Matthew Wicklund.;Miriam L Freimer.;Kate Eichinger.;Jeanne Dekdebrun.;Chao-Yin Chen.;Varun Goel.;Bradley McEvoy.;Yiming Zhu.;Steven G Hughes.;Elizabeth J Ackermann.;Arthur A Levin.
来源: N Engl J Med. 2026年394卷8期763-772页
Myotonic dystrophy type 1 is a rare, dominantly inherited, progressive, disabling, neuromuscular disease that leads to decreased life expectancy and has no approved therapies. The disease is caused by a trinucleotide repeat expansion in DMPK, which encodes myotonic dystrophy type 1 protein kinase and imparts a toxic gain of function to the transcribed messenger RNA (mRNA), resulting in dysregulated alternative splicing (missplicing). Delpacibart etedesiran (del-desiran [AOC 1001]) is a monoclonal antibody-oligonucleotide conjugate. The antibody component targets transferrin receptor 1, and the oligonucleotide component targets DMPK mRNA.
16. Hematopoietic Stem-Cell Gene Therapy for Cystinosis.
作者: Bruce A Barshop.;Edward D Ball.;Nadine Benador.;Doris Trauner.;Susan Phillips.;Ranjan Dohil.;Natalie A Afshari.;Sohini Roy.;Beatriz Campo Fernandes.;Donald Kohn.;Katayoon Shayan.;John K Everett.;Frederic D Bushman.;Julian Midgley.;Hong Liang.;Anne Sawyers.;Jon A Gangoiti.;Maulik Panchal.;Imama Ahmed.;Stephanie Cherqui.
来源: N Engl J Med. 2026年394卷8期753-762页
Cystinosis is a multisystemic lysosomal storage disorder caused by pathogenic variants in CTNS, the gene encoding cystinosin, a lysosomal transmembrane cystine transporter. In patients with cystinosis, cystine accumulates within lysosomes in all organs. The cystine-depleting agent cysteamine delays but does not prevent disease progression.
17. Dengue Suppression by Male Wolbachia-Infected Mosquitoes.
作者: Jue Tao Lim.;Chee-Seng Chong.;Chia-Chen Chang.;Diyar Mailepessov.;Borame Dickens.;Yee Ling Lai.;Lu Deng.;Caleb Lee.;Li Yun Tan.;Grace Chain.;Muhammad Faizal Zulkifli.;Jonathan Wee Kent Liew.;Kathryn Vasquez.;Man Ling Chau.;Youming Ng.;Vernon Lee.;Judith Chui Ching Wong.;Shuzhen Sim.;Cheong Huat Tan.;Lee Ching Ng.; .
来源: N Engl J Med. 2026年394卷12期1175-1183页
Wild-type female Aedes aegypti mosquitoes that mate with male A. aegypti mosquitoes that have been infected with the wAlbB strain of Wolbachia pipientis bacteria produce nonviable offspring owing to cytoplasmic incompatibility. Repeated releases of wolbachia-infected males can potentially suppress wild-type mosquito populations and reduce the risk of dengue virus infection.
18. AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.
作者: Connor J Lewis.;Precilla D'Souza.;Jean M Johnston.;Maria T Acosta.;Cristan Farmer.;Eva H Baker.;Anna Crowell.;Yoliann Mojica.;Sumaiya Ashraf.;Lisa Joseph.;Gilbert Vézina.;Zenaide Quezado.;Muhammad H Yousef.;Zeynep Vardar.;Mohammed Salman Shazeeb.;Manuela Corti.;Meghan Blackwood.;Kirsten Coleman.;Rita Batista.;Audrey Thurm.;Erika De Boever.;William A Gahl.;Barry J Byrne.;Terence R Flotte.;Xuntian Jiang.;Amanda L Gross.;Allison M Keeler.;Heather Gray-Edwards.;Douglas R Martin.;Miguel Sena-Esteves.;Cynthia J Tifft.
来源: N Engl J Med. 2026年394卷12期1184-1194页
GM1 gangliosidosis, caused by biallelic variants in GLB1, results from deficiency of lysosomal β-galactosidase, which degrades GM1 ganglioside. This fatal neurodegenerative disease currently has no effective therapy.
19. Palbociclib for Hormone-Receptor-Positive, HER2-Positive Advanced Breast Cancer.
作者: Otto Metzger.;Sumithra Mandrekar.;Shom Goel.;Joseph Gligorov.;Elgene Lim.;Eva Ciruelos.;Sibylle Loibl.;Travis Dockter.;Xavier Gonzàlez Farré.;Prudence A Francis.;Filipa Lynce.;Jane Lanzillotti.;Carter DuFrane.;Anna Wall.;Carrie Strand.;Ian Krop.;Ines Vaz-Luis.;Debu Tripathy.;Sherene Loi.;Aleix Prat.;Matthew Goetz.;Santiago Escrivá-de-Romaní.;David Porter.;Jennifer Spoenlein.;Daniel G Stover.;Sagar Sardesai.;Pierre Heudel.;Maria Koehler.;Cynthia Huang Bartlett.;Ariadna Holynskyj.;Prashanth Gopalakrishna.;Eric Gauthier.;Suzette Delaloge.;Kathy Miller.;Eric P Winer.;Luca Gianni.;Ann H Partridge.;Angela DeMichele.;Lisa A Carey.
来源: N Engl J Med. 2026年394卷5期451-462页
Dual anti-human epidermal growth factor receptor 2 (HER2) therapy plus chemotherapy followed by maintenance treatment with HER2-targeted and endocrine therapies is standard first-line treatment for hormone-receptor-positive, HER2-positive metastatic breast cancer. On the basis of preclinical and clinical data, the addition of palbociclib (a selective inhibitor of cyclin-dependent kinases 4 and 6) may overcome resistance to both endocrine and HER2-directed therapies.
20. A Randomized Trial of Tenecteplase in Acute Central Retinal Artery Occlusion.
作者: Stephen J Ryan.;Øystein Kalsnes Jørstad.;Mona Skjelland.;Maiju Pesonen.;Claus Z Simonsen.;Toke Bek.;Rolf Ankerlund Blauenfeldt.;Petra Ijäs.;Arja Laitinen.;Andrej Khanevski.;Jørgen Krohn.;Eyvind Rødahl.;Robin Lemmens.;Jelle Demeestere.;Catherine Cassiman.;Ingvild Nakstad.;Kristin Evensen.;Tiril Sandell.;Steffen Hamann.;Thomas C Truelsen.;Louisa M Christensen.;Sverre Rosenbaum.;Vaidas Matijošaitis.;Reda Žemaitienė.;Hanne Ellekjær.;Erlend Almaas.;Dordi Austeng.;Michael V Mazya.;Frank Traïsk.;Pauli Ylikotila.;Ulpu Salmi.;Kristian N Jenssen.;Håvard Lisether.;Cathrine Breivik.;Kristina Devik.;Lasse-Marius Elden Honningsvåg.;Jurgita Valaikienė.;Andrius Cimbalas.;Vetle Nilsen Malmberg.;Espen Anderson.;Ansar Roy.;Thor Håkon Skattør.;Kristian Lundsgaard Kraglund.;Christina Kefaloykos.;Inge Christoffer Olsen.;Peter Vanacker.;Daniel Strbian.;Morten C Moe.;Anne Hege Aamodt.; .
来源: N Engl J Med. 2026年394卷5期442-450页
Central retinal artery occlusion can result in permanent vision loss. Effective treatment is lacking.
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