Letters

  • Letter |

    Neuroimaging and cerebrospinal fluid analyses in humans reveal that loss of blood–brain barrier integrity and brain capillary pericyte damage are early biomarkers of cognitive impairment that occur independently of changes in amyloid-β and tau.

    • Daniel A. Nation
    • , Melanie D. Sweeney
    • , Axel Montagne
    • , Abhay P. Sagare
    • , Lina M. D’Orazio
    • , Maricarmen Pachicano
    • , Farshid Sepehrband
    • , Amy R. Nelson
    • , David P. Buennagel
    • , Michael G. Harrington
    • , Tammie L. S. Benzinger
    • , Anne M. Fagan
    • , John M. Ringman
    • , Lon S. Schneider
    • , John C. Morris
    • , Helena C. Chui
    • , Meng Law
    • , Arthur W. Toga
    •  & Berislav V. Zlokovic
  • Letter |

    A deep-learning algorithm, trained on over 17,000 real-world patient facial images, achieves high accuracy in identifying rare genetic disorders.

    • Yaron Gurovich
    • , Yair Hanani
    • , Omri Bar
    • , Guy Nadav
    • , Nicole Fleischer
    • , Dekel Gelbman
    • , Lina Basel-Salmon
    • , Peter M. Krawitz
    • , Susanne B. Kamphausen
    • , Martin Zenker
    • , Lynne M. Bird
    •  & Karen W. Gripp
  • Letter |

    A deep learning algorithm applied to the electrocardiogram—a test of the heart’s electrical activity—can detect abnormally low contractile function of the heart, opening up the possibility for a simple screening tool for this condition.

    • Zachi I. Attia
    • , Suraj Kapa
    • , Francisco Lopez-Jimenez
    • , Paul M. McKie
    • , Dorothy J. Ladewig
    • , Gaurav Satam
    • , Patricia A. Pellikka
    • , Maurice Enriquez-Sarano
    • , Peter A. Noseworthy
    • , Thomas M. Munger
    • , Samuel J. Asirvatham
    • , Christopher G. Scott
    • , Rickey E. Carter
    •  & Paul A. Friedman
  • Letter |

    The new RAF inhibitor PLX8394 selectively blocks ERK signaling in tumors driven by class 1 and/or class 2 BRAF mutations and BRAF fusions while maintaining a broad therapeutic window.

    • Zhan Yao
    • , Yijun Gao
    • , Wenjing Su
    • , Rona Yaeger
    • , Jessica Tao
    • , Na Na
    • , Ying Zhang
    • , Chao Zhang
    • , Andrey Rymar
    • , Anthony Tao
    • , Neilawattie M. Timaul
    • , Rory Mcgriskin
    • , Nathaniel A. Outmezguine
    • , HuiYong Zhao
    • , Qing Chang
    • , Besnik Qeriqi
    • , Mariano Barbacid
    • , Elisa de Stanchina
    • , David M Hyman
    • , Gideon Bollag
    •  & Neal Rosen
  • Letter |

    RNA editing enzyme ADAR1 is a therapeutic vulnerability in tumors with constitutive cell-autonomous production of interferon-stimulated genes

    • Huayang Liu
    • , Javad Golji
    • , Lauren K. Brodeur
    • , Franklin S. Chung
    • , Julie T. Chen
    • , Rosalie S. deBeaumont
    • , Caroline P. Bullock
    • , Michael D. Jones
    • , Grainne Kerr
    • , Li Li
    • , Daniel P. Rakiec
    • , Michael R. Schlabach
    • , Sosathya Sovath
    • , Joseph D. Growney
    • , Raymond A. Pagliarini
    • , David A. Ruddy
    • , Kenzie D. MacIsaac
    • , Joshua M. Korn
    •  & E. Robert McDonald III
  • Letter |

    High-resolution sequencing of longitudinal patient samples reveals subclonal mutational diversity in the stem cell compartment driving parallel evolution patterns in acute myeloid leukemia progression.

    • Jiahao Chen
    • , Yun-Ruei Kao
    • , Daqian Sun
    • , Tihomira I. Todorova
    • , David Reynolds
    • , Swathi-Rao Narayanagari
    • , Cristina Montagna
    • , Britta Will
    • , Amit Verma
    •  & Ulrich Steidl
  • Letter |

    Intratumoral T cells that are capable of recognizing adjacent tumor tissue antigens can be exceedingly rare.

    • Wouter Scheper
    • , Sander Kelderman
    • , Lorenzo F. Fanchi
    • , Carsten Linnemann
    • , Gavin Bendle
    • , Marije A. J. de Rooij
    • , Christian Hirt
    • , Riccardo Mezzadra
    • , Maarten Slagter
    • , Krijn Dijkstra
    • , Roelof J. C. Kluin
    • , Petur Snaebjornsson
    • , Katy Milne
    • , Brad H. Nelson
    • , Henry Zijlmans
    • , Gemma Kenter
    • , Emile E. Voest
    • , John B. A. G. Haanen
    •  & Ton N. Schumacher
  • Letter |

    Nongenetic activation of Aurora kinase A in the majority of patients with non-small-cell lung cancer mediates adaptive resistance to EGFR inhibition and offers an opportunity for combination treatment.

    • Khyati N. Shah
    • , Roma Bhatt
    • , Julia Rotow
    • , Julia Rohrberg
    • , Victor Olivas
    • , Victoria E. Wang
    • , Golzar Hemmati
    • , Maria M. Martins
    • , Ashley Maynard
    • , Jonathan Kuhn
    • , Jacqueline Galeas
    • , Hayley J. Donnella
    • , Swati Kaushik
    • , Angel Ku
    • , Sophie Dumont
    • , Gregor Krings
    • , Henry J. Haringsma
    • , Liliane Robillard
    • , Andrew D. Simmons
    • , Thomas C. Harding
    • , Frank McCormick
    • , Andrei Goga
    • , Collin M. Blakely
    • , Trever G. Bivona
    •  & Sourav Bandyopadhyay
  • Letter |

    Initial results from a first-in-human study show that PET imaging with PD-L1 antibodies outperforms immunohistochemistry- or RNA-sequencing-based biomarkers for prediction of clinical response to immunotherapy.

    • Frederike Bensch
    • , Elly L. van der Veen
    • , Marjolijn N. Lub-de Hooge
    • , Annelies Jorritsma-Smit
    • , Ronald Boellaard
    • , Iris C. Kok
    • , Sjoukje F. Oosting
    • , Carolina P. Schröder
    • , T. Jeroen N. Hiltermann
    • , Anthonie J. van der Wekken
    • , Harry J. M. Groen
    • , Thomas C. Kwee
    • , Sjoerd G. Elias
    • , Jourik A. Gietema
    • , Sandra Sanabria Bohorquez
    • , Alex de Crespigny
    • , Simon-Peter Williams
    • , Christoph Mancao
    • , Adrienne H. Brouwers
    • , Bernard M. Fine
    •  & Elisabeth G. E. de Vries
  • Letter |

    A new population of T helper cells employs unique signals to support autoreactive B cells in patients with systemic lupus erythematosus.

    • Simone Caielli
    • , Diogo Troggian Veiga
    • , Preetha Balasubramanian
    • , Shruti Athale
    • , Bojana Domic
    • , Elise Murat
    • , Romain Banchereau
    • , Zhaohui Xu
    • , Manjari Chandra
    • , Cheng-Han Chung
    • , Lynnette Walters
    • , Jeanine Baisch
    • , Tracey Wright
    • , Marilynn Punaro
    • , Lorien Nassi
    • , Katie Stewart
    • , Julie Fuller
    • , Duygu Ucar
    • , Hideki Ueno
    • , Joseph Zhou
    • , Jacques Banchereau
    •  & Virginia Pascual
  • Letter |

    Single-cell RNA-seq of a mouse model of multiple sclerosis uncovers new oligodendrocyte populations and putative disease markers and suggests new mechanisms underlying the pathogenesis of the disease.

    • Ana Mendanha Falcão
    • , David van Bruggen
    • , Sueli Marques
    • , Mandy Meijer
    • , Sarah Jäkel
    • , Eneritz Agirre
    • , Samudyata
    • , Elisa M. Floriddia
    • , Darya P. Vanichkina
    • , Charles ffrench-Constant
    • , Anna Williams
    • , André Ortlieb Guerreiro-Cacais
    •  & Gonçalo Castelo-Branco
  • Letter |

    Skin from individuals with a rare immunodeficiency harbors more eukaryotic viruses than healthy skin, highlighting the role of immune surveillance in modulating the skin microbiome.

    • Osnat Tirosh
    • , Sean Conlan
    • , Clay Deming
    • , Shih-Queen Lee-Lin
    • , Xin Huang
    • , Beatrice B. Barnabas
    • , Gerard G. Bouffard
    • , Shelise Y. Brooks
    • , Holly Marfani
    • , Lyudmila Dekhtyar
    • , Xiaobin Guan
    • , Joel Han
    • , Shi-ling Ho
    • , Richelle Legaspi
    • , Quino L. Maduro
    • , Catherine A. Masiello
    • , Jennifer C. McDowell
    • , Casandra Montemayor
    • , James C. Mullikin
    • , Morgan Park
    • , Nancy L. Riebow
    • , Karen Schandler
    • , Chanthra Scharer
    • , Brian Schmidt
    • , Christina Sison
    • , Sirintorn Stantripop
    • , James W. Thomas
    • , Pamela J. Thomas
    • , Meghana Vemulapalli
    • , Alice C. Young
    • , Helen C. Su
    • , Alexandra F. Freeman
    • , Julia A. Segre
    •  & Heidi H. Kong
  • Letter |

    Radiotherapy-induced abscopal responses enhance the efficacy of anti-CTLA-4 in patients with non-small-cell lung cancer.

    • Silvia C. Formenti
    • , Nils-Petter Rudqvist
    • , Encouse Golden
    • , Benjamin Cooper
    • , Erik Wennerberg
    • , Claire Lhuillier
    • , Claire Vanpouille-Box
    • , Kent Friedman
    • , Lucas Ferrari de Andrade
    • , Kai W. Wucherpfennig
    • , Adriana Heguy
    • , Naoko Imai
    • , Sacha Gnjatic
    • , Ryan O. Emerson
    • , Xi Kathy Zhou
    • , Tuo Zhang
    • , Abraham Chachoua
    •  & Sandra Demaria
  • Letter |

    AAV-mediated base editing corrects an autosomal recessive mutation in the Pahenu2 gene and ameliorates molecular deficits in a mouse model of metabolic liver disease.

    • Lukas Villiger
    • , Hiu Man Grisch-Chan
    • , Helen Lindsay
    • , Femke Ringnalda
    • , Chiara B. Pogliano
    • , Gabriella Allegri
    • , Ralph Fingerhut
    • , Johannes Häberle
    • , Joao Matos
    • , Mark D. Robinson
    • , Beat Thöny
    •  & Gerald Schwank
  • Letter |

    A biocompatible device built from naturally dissolving components and controlled by wireless technology enables programmable electrical stimulation of injured rodent peripheral nerves to accelerate regeneration and recovery.

    • Jahyun Koo
    • , Matthew R. MacEwan
    • , Seung-Kyun Kang
    • , Sang Min Won
    • , Manu Stephen
    • , Paul Gamble
    • , Zhaoqian Xie
    • , Ying Yan
    • , Yu-Yu Chen
    • , Jiho Shin
    • , Nathan Birenbaum
    • , Sangjin Chung
    • , Sung Bong Kim
    • , Jawad Khalifeh
    • , Daniel V. Harburg
    • , Kelsey Bean
    • , Michael Paskett
    • , Jeonghyun Kim
    • , Zohny S. Zohny
    • , Seung Min Lee
    • , Ruoyao Zhang
    • , Kaijing Luo
    • , Bowen Ji
    • , Anthony Banks
    • , Hyuck Mo Lee
    • , Younggang Huang
    • , Wilson Z. Ray
    •  & John A. Rogers
  • Letter |

    Viral-mediated base editing in utero enables therapeutic editing of two metabolic genes in mice.

    • Avery C. Rossidis
    • , John D. Stratigis
    • , Alexandra C. Chadwick
    • , Heather A. Hartman
    • , Nicholas J. Ahn
    • , Haiying Li
    • , Kshitiz Singh
    • , Barbara E. Coons
    • , Li Li
    • , Wenjian Lv
    • , Philip W. Zoltick
    • , Deepthi Alapati
    • , William Zacharias
    • , Rajan Jain
    • , Edward E. Morrisey
    • , Kiran Musunuru
    •  & William H. Peranteau
  • Letter |

    Neoadjuvant combination treatment with nivolumab and ipilimumab in patients with high-risk melanoma results in higher response rates than nivolumab monotherapy and warrants future optimization of dosing regimens to preserve efficacy while limiting toxicity.

    • Rodabe N. Amaria
    • , Sangeetha M. Reddy
    • , Hussein A. Tawbi
    • , Michael A. Davies
    • , Merrick I. Ross
    • , Isabella C. Glitza
    • , Janice N. Cormier
    • , Carol Lewis
    • , Wen-Jen Hwu
    • , Ehab Hanna
    • , Adi Diab
    • , Michael K. Wong
    • , Richard Royal
    • , Neil Gross
    • , Randal Weber
    • , Stephen Y. Lai
    • , Richard Ehlers
    • , Jorge Blando
    • , Denái R. Milton
    • , Scott Woodman
    • , Robin Kageyama
    • , Daniel K. Wells
    • , Patrick Hwu
    • , Sapna P. Patel
    • , Anthony Lucci
    • , Amy Hessel
    • , Jeffrey E. Lee
    • , Jeffrey Gershenwald
    • , Lauren Simpson
    • , Elizabeth M. Burton
    • , Liberty Posada
    • , Lauren Haydu
    • , Linghua Wang
    • , Shaojun Zhang
    • , Alexander J. Lazar
    • , Courtney W. Hudgens
    • , Vancheswaran Gopalakrishnan
    • , Alexandre Reuben
    • , Miles C. Andrews
    • , Christine N. Spencer
    • , Victor Prieto
    • , Padmanee Sharma
    • , James Allison
    • , Michael T. Tetzlaff
    •  & Jennifer A. Wargo
  • Letter |

    The long-term follow-up results of a phase 1/2 retinal gene therapy clinical trial for choroideremia ( NCT01461213 ) support the safety and efficacy of the treatment.

    • Kanmin Xue
    • , Jasleen K Jolly
    • , Alun R. Barnard
    • , Anna Rudenko
    • , Anna P. Salvetti
    • , Maria I. Patrício
    • , Thomas L. Edwards
    • , Markus Groppe
    • , Harry O. Orlans
    • , Tanya Tolmachova
    • , Graeme C. Black
    • , Andrew R. Webster
    • , Andrew J. Lotery
    • , Graham E. Holder
    • , Susan M. Downes
    • , Miguel C. Seabra
    •  & Robert E. MacLaren
  • Letter |

    Large tumors induce anemia and expansion of CD45+ immature erythroid cells, which represent a major immunosuppressive population in the spleen, contributing to systemic suppression of T cell immunity in late-stage cancer.

    • Lintao Zhao
    • , Ran He
    • , Haixia Long
    • , Bo Guo
    • , Qingzhu Jia
    • , Diyuan Qin
    • , Si-Qi Liu
    • , Zhongyu Wang
    • , Tong Xiang
    • , Jue Zhang
    • , Yulong Tan
    • , Jiani Huang
    • , Junying Chen
    • , Fang Wang
    • , Minglu Xiao
    • , Jianbao Gao
    • , Xinxin Yang
    • , Hao Zeng
    • , Xinxin Wang
    • , Chunyan Hu
    • , Peter B. Alexander
    • , Alistair L. J. Symonds
    • , Jia Yu
    • , Yisong Wan
    • , Qi-Jing Li
    • , Lilin Ye
    •  & Bo Zhu
  • Letter |

    Neoadjuvant combination immunotherapy in patients with advanced melanoma shows favorable activity over adjuvant treatment and warrants future evaluation with modified dosing schedules to reduce treatment-related adverse events.

    • Christian U. Blank
    • , Elisa A. Rozeman
    • , Lorenzo F. Fanchi
    • , Karolina Sikorska
    • , Bart van de Wiel
    • , Pia Kvistborg
    • , Oscar Krijgsman
    • , Marlous van den Braber
    • , Daisy Philips
    • , Annegien Broeks
    • , Johannes V. van Thienen
    • , Henk A. Mallo
    • , Sandra Adriaansz
    • , Sylvia ter Meulen
    • , Loes M. Pronk
    • , Lindsay G. Grijpink-Ongering
    • , Annemarie Bruining
    • , Rachel M. Gittelman
    • , Sarah Warren
    • , Harm van Tinteren
    • , Daniel S. Peeper
    • , John B. A. G. Haanen
    • , Alexander C. J. van Akkooi
    •  & Ton N. Schumacher
  • Letter |

    In vivo tracking of hematopoietic stem and progenitor cells in humans treated with lentiviral gene therapy unveils the resilience of hematopoietic stem cells and the long-term survival of lymphoid-biased precursors.

    • Serena Scala
    • , Luca Basso-Ricci
    • , Francesca Dionisio
    • , Danilo Pellin
    • , Stefania Giannelli
    • , Federica Andrea Salerio
    • , Lorena Leonardelli
    • , Maria Pia Cicalese
    • , Francesca Ferrua
    • , Alessandro Aiuti
    •  & Luca Biasco
  • Letter |

    Combination of two broadly neutralizing antibodies is effective in reducing HIV-1 viremia and in limiting the emergence of resistant viral variants in individuals harboring antibody-sensitive viruses.

    • Yotam Bar-On
    • , Henning Gruell
    • , Till Schoofs
    • , Joy A. Pai
    • , Lilian Nogueira
    • , Allison L. Butler
    • , Katrina Millard
    • , Clara Lehmann
    • , Isabelle Suárez
    • , Thiago Y. Oliveira
    • , Theodora Karagounis
    • , Yehuda Z. Cohen
    • , Christoph Wyen
    • , Stefan Scholten
    • , Lisa Handl
    • , Shiraz Belblidia
    • , Juan P. Dizon
    • , Jörg J. Vehreschild
    • , Maggi Witmer-Pack
    • , Irina Shimeliovich
    • , Kanika Jain
    • , Kerstin Fiddike
    • , Kelly E. Seaton
    • , Nicole L. Yates
    • , Jill Horowitz
    • , Roy M. Gulick
    • , Nico Pfeifer
    • , Georgia D. Tomaras
    • , Michael S. Seaman
    • , Gerd Fätkenheuer
    • , Marina Caskey
    • , Florian Klein
    •  & Michel C. Nussenzweig
  • Letter |

    Mitochondrially targeted zinc-finger nucleases reduce mutational burden and correct biochemical defects in a mouse model of mitochondrial disease.

    • Payam A. Gammage
    • , Carlo Viscomi
    • , Marie-Lune Simard
    • , Ana S. H. Costa
    • , Edoardo Gaude
    • , Christopher A. Powell
    • , Lindsey Van Haute
    • , Beverly J. McCann
    • , Pedro Rebelo-Guiomar
    • , Raffaele Cerutti
    • , Lei Zhang
    • , Edward J. Rebar
    • , Massimo Zeviani
    • , Christian Frezza
    • , James B. Stewart
    •  & Michal Minczuk
  • Letter |

    Intracortical activity data recorded over 2 years in a tetraplegic patient is used to develop an artificial intelligence algorithm that achieves fast, accurate, and stable movement decoding to reenable real-time control of the paralyzed forearm.

    • Michael A. Schwemmer
    • , Nicholas D. Skomrock
    • , Per B. Sederberg
    • , Jordyn E. Ting
    • , Gaurav Sharma
    • , Marcia A. Bockbrader
    •  & David A. Friedenberg
  • Letter |

    In a human subject with chronic paraplegia, a combination of epidural electrical stimulation and long-term rehabilitative training have culminated in the first report of unassisted, voluntary independent stepping in a paralyzed individual.

    • Megan L. Gill
    • , Peter J. Grahn
    • , Jonathan S. Calvert
    • , Margaux B. Linde
    • , Igor A. Lavrov
    • , Jeffrey A. Strommen
    • , Lisa A. Beck
    • , Dimitry G. Sayenko
    • , Meegan G. Van Straaten
    • , Dina I. Drubach
    • , Daniel D. Veith
    • , Andrew R. Thoreson
    • , Cesar Lopez
    • , Yury P. Gerasimenko
    • , V. Reggie Edgerton
    • , Kendall H. Lee
    •  & Kristin D. Zhao
  • Letter |

    In pediatric brain tumors that are accompanied by epileptic seizures, the BRAF somatic mutation V600E contributes to intrinsic epileptic properties in neurons, which can be suppressed by vemurafenib in mice.

    • Hyun Yong Koh
    • , Se Hoon Kim
    • , Jaeson Jang
    • , Hyungguk Kim
    • , Sungwook Han
    • , Jae Seok Lim
    • , Geurim Son
    • , Junjeong Choi
    • , Byung Ouk Park
    • , Won Do Heo
    • , Jinju Han
    • , Hyunjoo Jenny Lee
    • , Daeyoup Lee
    • , Hoon-Chul Kang
    • , Minho Shong
    • , Se-Bum Paik
    • , Dong Seok Kim
    •  & Jeong Ho Lee
  • Letter |

    The definition of a 'healthy' microbiome is impacted by geographic regional variations.

    • Yan He
    • , Wei Wu
    • , Hui-Min Zheng
    • , Pan Li
    • , Daniel McDonald
    • , Hua-Fang Sheng
    • , Mu-Xuan Chen
    • , Zi-Hui Chen
    • , Gui-Yuan Ji
    • , Zhong-Dai-Xi Zheng
    • , Prabhakar Mujagond
    • , Xiao-Jiao Chen
    • , Zu-Hua Rong
    • , Peng Chen
    • , Li-Yi Lyu
    • , Xian Wang
    • , Chong-Bin Wu
    • , Nan Yu
    • , Yan-Jun Xu
    • , Jia Yin
    • , Jeroen Raes
    • , Rob Knight
    • , Wen-Jun Ma
    •  & Hong-Wei Zhou
  • Letter |

    Stool microbiota composition correlates with the ethnic backgrounds of people living in the same city, suggesting that geographical location and ethnicity have distinct effects on microbiota.

    • Mélanie Deschasaux
    • , Kristien E. Bouter
    • , Andrei Prodan
    • , Evgeni Levin
    • , Albert K. Groen
    • , Hilde Herrema
    • , Valentina Tremaroli
    • , Guido J. Bakker
    • , Ilias Attaye
    • , Sara-Joan Pinto-Sietsma
    • , Daniel H. van Raalte
    • , Marieke B. Snijder
    • , Mary Nicolaou
    • , Ron Peters
    • , Aeilko H. Zwinderman
    • , Fredrik Bäckhed
    •  & Max Nieuwdorp
  • Letter |

    A gene signature identified in spontaneously regressing neuroblastoma identifies responders to immune checkpoint blockade among patients with melanoma with accuracy superior to previously reported biomarkers.

    • Noam Auslander
    • , Gao Zhang
    • , Joo Sang Lee
    • , Dennie T. Frederick
    • , Benchun Miao
    • , Tabea Moll
    • , Tian Tian
    • , Zhi Wei
    • , Sanna Madan
    • , Ryan J. Sullivan
    • , Genevieve Boland
    • , Keith Flaherty
    • , Meenhard Herlyn
    •  & Eytan Ruppin
  • Letter |

    A deep-learning algorithm is developed to provide rapid and accurate diagnosis of clinical 3D head CT-scan images to triage and prioritize urgent neurological events, thus potentially accelerating time to diagnosis and care in clinical settings.

    • Joseph J. Titano
    • , Marcus Badgeley
    • , Javin Schefflein
    • , Margaret Pain
    • , Andres Su
    • , Michael Cai
    • , Nathaniel Swinburne
    • , John Zech
    • , Jun Kim
    • , Joshua Bederson
    • , J. Mocco
    • , Burton Drayer
    • , Joseph Lehar
    • , Samuel Cho
    • , Anthony Costa
    •  & Eric K. Oermann
  • Letter |

    T cells kill blood-stage Plasmodium vivax, providing a rationale for the development of a T cell vaccine against this parasite.

    • Caroline Junqueira
    • , Camila R. R. Barbosa
    • , Pedro A. C. Costa
    • , Andréa Teixeira-Carvalho
    • , Guilherme Castro
    • , Sumit Sen Santara
    • , Rafael P. Barbosa
    • , Farokh Dotiwala
    • , Dhelio B. Pereira
    • , Lis R. Antonelli
    • , Judy Lieberman
    •  & Ricardo T. Gazzinelli
  • Letter |

    Retroelements located in antisense orientation within interferon-regulated genes are reactivated in a subset of cancer cells and initiate a STING- and MAVS-dependent feed-forward inflammatory loop, driving antitumor immunity and exhaustion.

    • Israel Cañadas
    • , Rohit Thummalapalli
    • , Jong Wook Kim
    • , Shunsuke Kitajima
    • , Russell William Jenkins
    • , Camilla Laulund Christensen
    • , Marco Campisi
    • , Yanan Kuang
    • , Yanxi Zhang
    • , Evisa Gjini
    • , Gao Zhang
    • , Tian Tian
    • , Debattama Rai. Sen
    • , Diana Miao
    • , Yu Imamura
    • , Tran Thai
    • , Brandon Piel
    • , Hideki Terai
    • , Amir Reza Aref
    • , Timothy Hagan
    • , Shohei Koyama
    • , Masayuki Watanabe
    • , Hideo Baba
    • , Anika Elise Adeni
    • , Christine Anne Lydon
    • , Pablo Tamayo
    • , Zhi Wei
    • , Meenhard Herlyn
    • , Thanh Uyen Barbie
    • , Ravindra Uppaluri
    • , Lynnette Marie Sholl
    • , Ewa Sicinska
    • , Jacob Sands
    • , Scott Rodig
    • , Kwok Kin Wong
    • , Cloud Peter Paweletz
    • , Hideo Watanabe
    •  & David Allen Barbie
  • Letter |

    In utero GBA gene therapy extends lifespan and provides long-lasting phenotypic amelioration in a mouse model of neuronopathic Gaucher disease. Fetal ultrasound-guided in utero gene vector delivery is also achieved in the non-human primate brain.

    • Giulia Massaro
    • , Citra N. Z. Mattar
    • , Andrew M. S. Wong
    • , Ernestas Sirka
    • , Suzanne M. K. Buckley
    • , Bronwen R. Herbert
    • , Stefan Karlsson
    • , Dany P. Perocheau
    • , Derek Burke
    • , Simon Heales
    • , Angela Richard-Londt
    • , Sebastian Brandner
    • , Mylene Huebecker
    • , David A. Priestman
    • , Frances M. Platt
    • , Kevin Mills
    • , Arijit Biswas
    • , Jonathan D. Cooper
    • , Jerry K. Y. Chan
    • , Seng H. Cheng
    • , Simon N. Waddington
    •  & Ahad A. Rahim
  • Letter |

    A glutamate-gated chloride channel delivered via gene therapy is shown to detect elevated brain glutamate levels and trigger the suppression of neuronal excitability, thereby attenuating seizure activity in two rodent models of epilepsy.

    • Andreas Lieb
    • , Yichen Qiu
    • , Christine L. Dixon
    • , Janosch P. Heller
    • , Matthew C. Walker
    • , Stephanie Schorge
    •  & Dimitri M. Kullmann
  • Letter |

    Targeting a post-translational modification of Fas by recombinant Glrx reverses established lung fibrosis in a mouse model of age-related idiopathic pulmonary fibrosis.

    • Vikas Anathy
    • , Karolyn G. Lahue
    • , David G. Chapman
    • , Shi B. Chia
    • , Dylan T. Casey
    • , Reem Aboushousha
    • , Jos L. J. van der Velden
    • , Evan Elko
    • , Sidra M. Hoffman
    • , David H. McMillan
    • , Jane T. Jones
    • , James D. Nolin
    • , Sarah Abdalla
    • , Robert Schneider
    • , David J. Seward
    • , Elle C. Roberson
    • , Matthew D. Liptak
    • , Morgan E. Cousins
    • , Kelly J. Butnor
    • , Douglas. J. Taatjes
    • , Ralph C. Budd
    • , Charles G. Irvin
    • , Ye-Shih Ho
    • , Razq Hakem
    • , Kevin K. Brown
    • , Reiko Matsui
    • , Markus M. Bachschmid
    • , Jose L. Gomez
    • , Naftali Kaminski
    • , Albert van der Vliet
    •  & Yvonne M. W. Janssen-Heininger
  • Letter |

    Small-molecule inhibitors of the serine dipeptidases DPP8 and DPP9 block AML progression by promoting CARD8-dependent pyroptosis of leukemic myeloid cells.

    • Darren C. Johnson
    • , Cornelius Y. Taabazuing
    • , Marian C. Okondo
    • , Ashley J. Chui
    • , Sahana D. Rao
    • , Fiona C. Brown
    • , Casie Reed
    • , Elizabeth Peguero
    • , Elisa de Stanchina
    • , Alex Kentsis
    •  & Daniel A. Bachovchin
  • Letter |

    Metformin reverses established lung fibrosis in a bleomycin model in mice.

    • Sunad Rangarajan
    • , Nathaniel B. Bone
    • , Anna A. Zmijewska
    • , Shaoning Jiang
    • , Dae Won Park
    • , Karen Bernard
    • , Morgan L. Locy
    • , Saranya Ravi
    • , Jessy Deshane
    • , Roslyn B. Mannon
    • , Edward Abraham
    • , Victor Darley-Usmar
    • , Victor J. Thannickal
    •  & Jaroslaw W. Zmijewski
  • Letter |

    Extensive, high-dimensional characterization of T cells in breast cancer reveals activated TRM population and a gene signature associated with improved prognosis.

    • Peter Savas
    • , Balaji Virassamy
    • , Chengzhong Ye
    • , Agus Salim
    • , Christopher P. Mintoff
    • , Franco Caramia
    • , Roberto Salgado
    • , David J. Byrne
    • , Zhi L. Teo
    • , Sathana Dushyanthen
    • , Ann Byrne
    • , Lironne Wein
    • , Stephen J. Luen
    • , Catherine Poliness
    • , Sophie S. Nightingale
    • , Anita S. Skandarajah
    • , David E. Gyorki
    • , Chantel M. Thornton
    • , Paul A. Beavis
    • , Stephen B. Fox
    • , Phillip K. Darcy
    • , Terence P. Speed
    • , Laura K. Mackay
    • , Paul J. Neeson
    •  & Sherene Loi
  • Letter |

    Indoleamine 2,3-dioxygenase normally suppresses inflammation, but knockout of its gene is metabolically beneficial as its depletion reshapes the gut microbiota.

    • Ludivine Laurans
    • , Nicolas Venteclef
    • , Yacine Haddad
    • , Mouna Chajadine
    • , Fawaz Alzaid
    • , Sarvenaz Metghalchi
    • , Bruno Sovran
    • , Raphael G. P. Denis
    • , Julien Dairou
    • , Marina Cardellini
    • , Jose-Maria Moreno-Navarrete
    • , Marjolene Straub
    • , Sarah Jegou
    • , Claire McQuitty
    • , Thomas Viel
    • , Bruno Esposito
    • , Bertrand Tavitian
    • , Jacques Callebert
    • , Serge H. Luquet
    • , Massimo Federici
    • , José Manuel Fernandez-Real
    • , Remy Burcelin
    • , Jean-Marie Launay
    • , Alain Tedgui
    • , Ziad Mallat
    • , Harry Sokol
    •  & Soraya Taleb
  • Letter |

    Deep single-cell RNA sequencing of tumor-infiltrating T cells in non-small-cell lung cancer identifies features associated with functional states and clinical outcome

    • Xinyi Guo
    • , Yuanyuan Zhang
    • , Liangtao Zheng
    • , Chunhong Zheng
    • , Jintao Song
    • , Qiming Zhang
    • , Boxi Kang
    • , Zhouzerui Liu
    • , Liang Jin
    • , Rui Xing
    • , Ranran Gao
    • , Lei Zhang
    • , Minghui Dong
    • , Xueda Hu
    • , Xianwen Ren
    • , Dennis Kirchhoff
    • , Helge Gottfried Roider
    • , Tiansheng Yan
    •  & Zemin Zhang
  • Letter |

    ALS/FTD-related C9orf72 dipeptide-repeat proteins inhibit protein translation and impair stress granule dynamics, and they cause motor and cognitive deficits in mice.

    • Yong-Jie Zhang
    • , Tania F. Gendron
    • , Mark T. W. Ebbert
    • , Aliesha D. O’Raw
    • , Mei Yue
    • , Karen Jansen-West
    • , Xu Zhang
    • , Mercedes Prudencio
    • , Jeannie Chew
    • , Casey N. Cook
    • , Lillian M. Daughrity
    • , Jimei Tong
    • , Yuping Song
    • , Sarah R. Pickles
    • , Monica Castanedes-Casey
    • , Aishe Kurti
    • , Rosa Rademakers
    • , Bjorn Oskarsson
    • , Dennis W. Dickson
    • , Wenqian Hu
    • , Aaron D. Gitler
    • , John D. Fryer
    •  & Leonard Petrucelli
  • Letter |

    Agonism of microglial glucagon-like peptide-1 receptor (GLP1R) using a brain-penetrant peptide prevents the generation of neurotoxic astrocytes and ameliorates disease progression in two rodent models of Parkinson’s disease.

    • Seung Pil Yun
    • , Tae-In Kam
    • , Nikhil Panicker
    • , SangMin Kim
    • , Yumin Oh
    • , Jong-Sung Park
    • , Seung-Hwan Kwon
    • , Yong Joo Park
    • , Senthilkumar S. Karuppagounder
    • , Hyejin Park
    • , Sangjune Kim
    • , Nayeon Oh
    • , Nayoung Alice Kim
    • , Saebom Lee
    • , Saurav Brahmachari
    • , Xiaobo Mao
    • , Jun Hee Lee
    • , Manoj Kumar
    • , Daniel An
    • , Sung-Ung Kang
    • , Yunjong Lee
    • , Kang Choon Lee
    • , Dong Hee Na
    • , Donghoon Kim
    • , Sang Hun Lee
    • , Viktor V. Roschke
    • , Shane A. Liddelow
    • , Zoltan Mari
    • , Ben A. Barres
    • , Valina L. Dawson
    • , Seulki Lee
    • , Ted M. Dawson
    •  & Han Seok Ko
  • Letter |

    In vivo shRNA screens in sensitized genetic backgrounds identify p53-activated target genes involved in DNA repair that enable its tumor suppressor function.

    • Ana Janic
    • , Liz J. Valente
    • , Matthew J. Wakefield
    • , Leon Di Stefano
    • , Liz Milla
    • , Stephen Wilcox
    • , Haoyu Yang
    • , Lin Tai
    • , Cassandra J. Vandenberg
    • , Andrew J. Kueh
    • , Shinsuke Mizutani
    • , Margs S. Brennan
    • , Robyn L. Schenk
    • , Lisa M. Lindqvist
    • , Anthony T. Papenfuss
    • , Liam O’Connor
    • , Andreas Strasser
    •  & Marco J. Herold