Brief Communications

  • Brief Communication |

    Inhibition of CSF-1R with recurrent activating alterations and other actionable targets identified by genomic sequencing elicits clinical activity in patients with histiocytosis.

    • Benjamin H. Durham
    • , Estibaliz Lopez Rodrigo
    • , Jennifer Picarsic
    • , David Abramson
    • , Veronica Rotemberg
    • , Steven De Munck
    • , Erwin Pannecoucke
    • , Sydney X. Lu
    • , Alessandro Pastore
    • , Akihide Yoshimi
    • , Diana Mandelker
    • , Ozge Ceyhan-Birsoy
    • , Gary A. Ulaner
    • , Michael Walsh
    • , Mariko Yabe
    • , Kseniya Petrova-Drus
    • , Maria E. Arcila
    • , Marc Ladanyi
    • , David B. Solit
    • , Michael F. Berger
    • , David M. Hyman
    • , Mario E. Lacouture
    • , Caroline Erickson
    • , Ruth Saganty
    • , Michelle Ki
    • , Ira J. Dunkel
    • , Vicente Santa-María López
    • , Jaume Mora
    • , Julien Haroche
    • , Jean-Francois Emile
    • , Olivier Decaux
    • , Frederic Geissmann
    • , Savvas N. Savvides
    • , Alexander Drilon
    • , Eli L. Diamond
    •  & Omar Abdel-Wahab
  • Brief Communication |

    A unique case from the Colombian cohort of autosomal dominant Alzheimer’s disease is reported in which disease progression is substantially delayed despite unusually high amyloid plaque pathology, possibly related to a rare mutation in APOE3.

    • Joseph F. Arboleda-Velasquez
    • , Francisco Lopera
    • , Michael O’Hare
    • , Santiago Delgado-Tirado
    • , Claudia Marino
    • , Natalia Chmielewska
    • , Kahira L. Saez-Torres
    • , Dhanesh Amarnani
    • , Aaron P. Schultz
    • , Reisa A. Sperling
    • , David Leyton-Cifuentes
    • , Kewei Chen
    • , Ana Baena
    • , David Aguillon
    • , Silvia Rios-Romenets
    • , Margarita Giraldo
    • , Edmarie Guzmán-Vélez
    • , Daniel J. Norton
    • , Enmanuelle Pardilla-Delgado
    • , Arabiye Artola
    • , Justin S. Sanchez
    • , Juliana Acosta-Uribe
    • , Matthew Lalli
    • , Kenneth S. Kosik
    • , Matthew J. Huentelman
    • , Henrik Zetterberg
    • , Kaj Blennow
    • , Rebecca A. Reiman
    • , Ji Luo
    • , Yinghua Chen
    • , Pradeep Thiyyagura
    • , Yi Su
    • , Gyungah R. Jun
    • , Marcus Naymik
    • , Xiaowu Gai
    • , Moiz Bootwalla
    • , Jianling Ji
    • , Lishuang Shen
    • , John B. Miller
    • , Leo A. Kim
    • , Pierre N. Tariot
    • , Keith A. Johnson
    • , Eric M. Reiman
    •  & Yakeel T. Quiroz
  • Brief Communication |

    This first-in-human study assessed the feasibility of vaginal microbiome transplantation from healthy donors as a therapeutic alternative for patients suffering from symptomatic, intractable and recurrent bacterial vaginosis.

    • Ahinoam Lev-Sagie
    • , Debra Goldman-Wohl
    • , Yotam Cohen
    • , Mally Dori-Bachash
    • , Avner Leshem
    • , Uria Mor
    • , Jacob Strahilevitz
    • , Allon E. Moses
    • , Hagit Shapiro
    • , Simcha Yagel
    •  & Eran Elinav
  • Brief Communication |

    A deep residual learning framework identifies microsatellite instability in histology slides from patients with cancer and can be used to guide immunotherapy.

    • Jakob Nikolas Kather
    • , Alexander T. Pearson
    • , Niels Halama
    • , Dirk Jäger
    • , Jeremias Krause
    • , Sven H. Loosen
    • , Alexander Marx
    • , Peter Boor
    • , Frank Tacke
    • , Ulf Peter Neumann
    • , Heike I. Grabsch
    • , Takaki Yoshikawa
    • , Hermann Brenner
    • , Jenny Chang-Claude
    • , Michael Hoffmeister
    • , Christian Trautwein
    •  & Tom Luedde
  • Brief Communication |

    Individuals homozygous for the CCR5-∆32 allele have a 21% increase in mortality rate in the UK Biobank cohort. In light of the CRISPR-baby scandal, this work highlights the need for understanding the unintended consequences of introducing mutations in humans.

    • Xinzhu Wei
    •  & Rasmus Nielsen
  • Brief Communication |

    Mass cytometry identifies a unique phenotype enriched in CD4+ T cells isolated from autoimmune disease patients.

    • Asbjørn Christophersen
    • , Eivind G. Lund
    • , Omri Snir
    • , Elsa Solà
    • , Chakravarthi Kanduri
    • , Shiva Dahal-Koirala
    • , Stephanie Zühlke
    • , Øyvind Molberg
    • , Paul J. Utz
    • , Mina Rohani-Pichavant
    • , Julia F. Simard
    • , Cornelia L. Dekker
    • , Knut E. A. Lundin
    • , Ludvig M. Sollid
    •  & Mark M. Davis
  • Brief Communication |

    AAV-mediated CRISPR/Cas9 therapy extends life span and ameliorates disease-related phenotypes in a mouse model of Hutchinson–Gilford progeria syndrome.

    • Olaya Santiago-Fernández
    • , Fernando G. Osorio
    • , Víctor Quesada
    • , Francisco Rodríguez
    • , Sammy Basso
    • , Daniel Maeso
    • , Loïc Rolas
    • , Anna Barkaway
    • , Sussan Nourshargh
    • , Alicia R. Folgueras
    • , José M. P. Freije
    •  & Carlos López-Otín
  • Brief Communication |

    An algorithm trained on half a million electronic medical records predicts chronic kidney disease in diabetic patients using a small set of defined clinical features, outperforming predictions derived from clinical trial data.

    • Stefan Ravizza
    • , Tony Huschto
    • , Anja Adamov
    • , Lars Böhm
    • , Alexander Büsser
    • , Frederik F. Flöther
    • , Rolf Hinzmann
    • , Helena König
    • , Scott M. McAhren
    • , Daniel H. Robertson
    • , Titus Schleyer
    • , Bernd Schneidinger
    •  & Wolfgang Petrich
  • Brief Communication |

    RNA antisense oligonucleotide therapy to restore normal splicing of a ciliopathy gene shows promising safety and efficacy results in a clinical trial to treat a form of childhood blindness.

    • Artur V. Cideciyan
    • , Samuel G. Jacobson
    • , Arlene V. Drack
    • , Allen C. Ho
    • , Jason Charng
    • , Alexandra V. Garafalo
    • , Alejandro J. Roman
    • , Alexander Sumaroka
    • , Ian C. Han
    • , Maria D. Hochstedler
    • , Wanda L. Pfeifer
    • , Elliott H. Sohn
    • , Magali Taiel
    • , Michael R. Schwartz
    • , Patricia Biasutto
    • , Wilma de Wit
    • , Michael E. Cheetham
    • , Peter Adamson
    • , David M. Rodman
    • , Gerard Platenburg
    • , Maria D. Tome
    • , Irina Balikova
    • , Fanny Nerinckx
    • , Julie De Zaeytijd
    • , Caroline Van Cauwenbergh
    • , Bart P. Leroy
    •  & Stephen R. Russell
  • Brief Communication |

    Mutational signatures in melanoma are associated with prognostic features in patients and suggest distinct disease etiologies associated with the influence of different wavelengths of ultraviolet radiation.

    • Lucas D. Trucco
    • , Piyushkumar A. Mundra
    • , Kate Hogan
    • , Pablo Garcia-Martinez
    • , Amaya Viros
    • , Amit K. Mandal
    • , Nicolas Macagno
    • , Caroline Gaudy-Marqueste
    • , Donald Allan
    • , Franziska Baenke
    • , Martin Cook
    • , Clare McManus
    • , Berta Sanchez-Laorden
    • , Nathalie Dhomen
    •  & Richard Marais
  • Brief Communication |

    Preliminary evidence from two cases suggests that fecal microbiota transplantation may provide a viable treatment option for a severe adverse effect of immune checkpoint blockade therapy in patients with cancer.

    • Yinghong Wang
    • , Diana H. Wiesnoski
    • , Beth A. Helmink
    • , Vancheswaran Gopalakrishnan
    • , Kati Choi
    • , Hebert L. DuPont
    • , Zhi-Dong Jiang
    • , Hamzah Abu-Sbeih
    • , Christopher A. Sanchez
    • , Chia-Chi Chang
    • , Edwin R. Parra
    • , Alejandro Francisco-Cruz
    • , Gottumukkala S. Raju
    • , John R. Stroehlein
    • , Matthew T. Campbell
    • , Jianjun Gao
    • , Sumit K. Subudhi
    • , Dipen M. Maru
    • , Jorge M. Blando
    • , Alexander J. Lazar
    • , James P. Allison
    • , Padmanee Sharma
    • , Michael T. Tetzlaff
    • , Jennifer A. Wargo
    •  & Robert R. Jenq
  • Brief Communication |

    A CAR gene unintentionally introduced in a contaminating leukemia cell during the manufacturing of CAR T cells caused a patient to relapse after therapy.

    • Marco Ruella
    • , Jun Xu
    • , David M. Barrett
    • , Joseph A. Fraietta
    • , Tyler J. Reich
    • , David E. Ambrose
    • , Michael Klichinsky
    • , Olga Shestova
    • , Prachi R. Patel
    • , Irina Kulikovskaya
    • , Farzana Nazimuddin
    • , Vijay G. Bhoj
    • , Elena J. Orlando
    • , Terry J. Fry
    • , Hans Bitter
    • , Shannon L. Maude
    • , Bruce L. Levine
    • , Christopher L. Nobles
    • , Frederic D. Bushman
    • , Regina M. Young
    • , John Scholler
    • , Saar I. Gill
    • , Carl H. June
    • , Stephan A. Grupp
    • , Simon F. Lacey
    •  & J. Joseph Melenhorst
  • Brief Communication |

    Mutations in the CD19 gene suggesting irreversible loss of its surface expression are identified in the majority of analyzed cases of CD19 relapse in two clinical trials of pediatric ALL CD19 CAR T therapy, offering considerations for the rational choice of follow-up therapies.

    • Elena J. Orlando
    • , Xia Han
    • , Catherine Tribouley
    • , Patricia A. Wood
    • , Rebecca J. Leary
    • , Markus Riester
    • , John E. Levine
    • , Muna Qayed
    • , Stephan A. Grupp
    • , Michael Boyer
    • , Barbara De Moerloose
    • , Eneida R. Nemecek
    • , Henrique Bittencourt
    • , Hidefumi Hiramatsu
    • , Jochen Buechner
    • , Stella M. Davies
    • , Michael R. Verneris
    • , Kevin Nguyen
    • , Jennifer L. Brogdon
    • , Hans Bitter
    • , Michael Morrissey
    • , Piotr Pierog
    • , Serafino Pantano
    • , Jeffrey A. Engelman
    •  & Wendy Winckler
  • Brief Communication |

    Antibiotic treatment of SIV-infected nonhuman primates shows that inducing bacterial dysbiosis in the gut—similar to that seen in HIV infection—does not promote disease progression, questioning its potential role in progression to HIV/AIDS.

    • Alexandra M. Ortiz
    • , Jacob K. Flynn
    • , Sarah R. DiNapoli
    • , Ivan Vujkovic-Cvijin
    • , Carly E. Starke
    • , Stephen H. Lai
    • , MacKenzie E. Long
    • , Ornella Sortino
    • , Carol L. Vinton
    • , Joseph C. Mudd
    • , Leslie Johnston
    • , Kathleen Busman-Sahay
    • , Yasmine Belkaid
    • , Jacob D. Estes
    •  & Jason M. Brenchley
  • Brief Communication |

    Zika virus infection during pregnancy is associated with an increased rate of fetal loss in nonhuman primates, as reported in this multicenter analysis.

    • Dawn M. Dudley
    • , Koen K. Van Rompay
    • , Lark L. Coffey
    • , Amir Ardeshir
    • , Rebekah I. Keesler
    • , Eliza Bliss-Moreau
    • , Peta L. Grigsby
    • , Rosemary J. Steinbach
    • , Alec J. Hirsch
    • , Rhonda P. MacAllister
    • , Heidi L. Pecoraro
    • , Lois M. Colgin
    • , Travis Hodge
    • , Daniel N. Streblow
    • , Suzette Tardif
    • , Jean L. Patterson
    • , Manasi Tamhankar
    • , Maxim Seferovic
    • , Kjersti M. Aagaard
    • , Claudia Sánchez-San Martín
    • , Charles Y. Chiu
    • , Antonito T. Panganiban
    • , Ronald S. Veazey
    • , Xiaolei Wang
    • , Nicholas J. Maness
    • , Margaret H. Gilbert
    • , Rudolf P. Bohm
    • , Kristina M. Adams Waldorf
    • , Michael Gale Jr
    • , Lakshmi Rajagopal
    • , Charlotte E. Hotchkiss
    • , Emma L. Mohr
    • , Saverio V. Capuano III
    • , Heather A. Simmons
    • , Andres Mejia
    • , Thomas C. Friedrich
    • , Thaddeus G. Golos
    •  & David H. O’Connor
  • Brief Communication |

    Initiation of antiretroviral therapy in the first 2 weeks of HIV infection fails to prevent resurgence of virus after stopping treatment, indicating early establishment of a resilient viral reservoir.

    • Donn J. Colby
    • , Lydie Trautmann
    • , Suteeraporn Pinyakorn
    • , Louise Leyre
    • , Amélie Pagliuzza
    • , Eugène Kroon
    • , Morgane Rolland
    • , Hiroshi Takata
    • , Supranee Buranapraditkun
    • , Jintana Intasan
    • , Nitiya Chomchey
    • , Roshell Muir
    • , Elias K. Haddad
    • , Sodsai Tovanabutra
    • , Sasiwimol Ubolyam
    • , Diane L. Bolton
    • , Brandie A. Fullmer
    • , Robert J. Gorelick
    • , Lawrence Fox
    • , Trevor A. Crowell
    • , Rapee Trichavaroj
    • , Robert O’Connell
    • , Nicolas Chomont
    • , Jerome H. Kim
    • , Nelson L. Michael
    • , Merlin L. Robb
    • , Nittaya Phanuphak
    • , Jintanat Ananworanich
    • , P. Phanuphak
    • , N. Teeratakulpisarn
    • , S. Chottanapund
    • , M. de Souza
    • , J. Fletcher
    • , P. Tantivitayakul
    • , P. Eamyoung
    • , D. Sutthichom
    • , P. Prueksakaew
    • , S. Puttamaswin
    • , S. Tipsuk
    • , K. Benjapornpong
    • , N. Ratnaratorn
    • , C. Munkong
    • , K. Tanjnareel
    • , R. Kanaprach
    • , R. Rerknimitr
    • , P. Wattanaboonyongcharoen
    • , P. Rojnuckarin
    • , S. Manasnayakorn
    • , A. Schuetz
    • , S. Akapirat
    • , B. Nuntapinit
    • , N. Tantibul
    • , H. Savadsuk
    • , Y. Phuang-Ngern
    • , S. Jongrakthaitae
    • , W. Chuenaron
    • , N. Churikanont
    • , S. Getchalarat
    • , S. Krebs
    • , B. Slike
    • , A. Tokarev
    • , E. Sanders-Buell
    • , M. Bose
    • , C. Ogega
    • , J. Buahen
    • , M. Ouellette
    • , C. McCullough
    • , O. Butterworth
    • , E. Turk
    • , L. A. Eller
    • , M. Milazzo
    • , J. Mitchell
    • , C. Subra
    • , N. Lima
    • , J. Garnett
    • , F. Fatmi
    • , A. Sy
    • , N. Dawson
    • , S. Spudich
    • , V. Valcour
    • , F. Maldarelli
    • , I. Sereti
    • , J. Lifson
    • , R. Paul
    • , R. Fromentin
    •  & P. Dawson
  • Brief Communication |

    Treatment with setmelanotide, a new-generation MC4R agonist, provides durable weight loss in hyperphagic, leptin receptor–deficient patients, suggesting a pharmacological avenue to treat patients with various MC4R pathway defects.

    • Karine Clément
    • , Heike Biebermann
    • , I. Sadaf Farooqi
    • , Lex Van der Ploeg
    • , Barbara Wolters
    • , Christine Poitou
    • , Lia Puder
    • , Fred Fiedorek
    • , Keith Gottesdiener
    • , Gunnar Kleinau
    • , Nicolas Heyder
    • , Patrick Scheerer
    • , Ulrike Blume-Peytavi
    • , Irina Jahnke
    • , Shubh Sharma
    • , Jacek Mokrosinski
    • , Susanna Wiegand
    • , Anne Müller
    • , Katja Weiß
    • , Knut Mai
    • , Joachim Spranger
    • , Annette Grüters
    • , Oliver Blankenstein
    • , Heiko Krude
    •  & Peter Kühnen
  • Brief Communication |

    Thomas Geisbert and colleagues show that a cocktail of monoclonal antibodies protects cynomolgus monkeys from lethal Lassa fever virus infection, including when administration is delayed by more than a week after viral challenge.

    • Chad E Mire
    • , Robert W Cross
    • , Joan B Geisbert
    • , Viktoriya Borisevich
    • , Krystle N Agans
    • , Daniel J Deer
    • , Megan L Heinrich
    • , Megan M Rowland
    • , Augustine Goba
    • , Mambu Momoh
    • , Mathew L Boisen
    • , Donald S Grant
    • , Mohamed Fullah
    • , Sheik Humarr Khan
    • , Karla A Fenton
    • , James E Robinson
    • , Luis M Branco
    • , Robert F Garry
    •  & Thomas W Geisbert
  • Brief Communication |

    Bispecific antibodies that connect T cells with tumor cells can be delivered in the form of in vitro–transcribed pharmacologically optimized mRNA; when injected into mice, these mRNA-encoded antibodies reject large established tumors as efficiently as the corresponding recombinant antibody protein.

    • Christiane R Stadler
    • , Hayat Bähr-Mahmud
    • , Leyla Celik
    • , Bernhard Hebich
    • , Alexandra S Roth
    • , René P Roth
    • , Katalin Karikó
    • , Özlem Türeci
    •  & Ugur Sahin
  • Brief Communication |

    Metformin treatment for 10 d rescues several behavioral deficits related to fragile X syndrome in adult Fmr1−/y mice.

    • Ilse Gantois
    • , Arkady Khoutorsky
    • , Jelena Popic
    • , Argel Aguilar-Valles
    • , Erika Freemantle
    • , Ruifeng Cao
    • , Vijendra Sharma
    • , Tine Pooters
    • , Anmol Nagpal
    • , Agnieszka Skalecka
    • , Vinh T Truong
    • , Shane Wiebe
    • , Isabelle A Groves
    • , Seyed Mehdi Jafarnejad
    • , Clément Chapat
    • , Elizabeth A McCullagh
    • , Karine Gamache
    • , Karim Nader
    • , Jean-Claude Lacaille
    • , Christos G Gkogkas
    •  & Nahum Sonenberg
  • Brief Communication |

    Prostate cancer is refractory to anti-CTLA-4 therapy, but the reason why is unclear. Padmanee Sharma and colleagues report that the inhibitory molecule VISTA, which negatively regulates T cells, is upregulated on macrophages in prostate tumors that have been treated with anti-CTLA-4 and may play a role in resistance to this immunotherapy.

    • Jianjun Gao
    • , John F Ward
    • , Curtis A Pettaway
    • , Lewis Z Shi
    • , Sumit K Subudhi
    • , Luis M Vence
    • , Hao Zhao
    • , Jianfeng Chen
    • , Hong Chen
    • , Eleni Efstathiou
    • , Patricia Troncoso
    • , James P Allison
    • , Christopher J Logothetis
    • , Ignacio I Wistuba
    • , Manuel A Sepulveda
    • , Jingjing Sun
    • , Jennifer Wargo
    • , Jorge Blando
    •  & Padmanee Sharma
  • Brief Communication |

    Antibody-based diagnosis of Zika virus (ZIKV) infection is complicated by cross-reactivity with antibodies against dengue virus. Hongjie Dai and colleagues report their development of a new IgA- and IgG-based diagnostic test that detects ZIKV with high specificity.

    • Bo Zhang
    • , Benjamin A Pinsky
    • , Jeyarama S Ananta
    • , Su Zhao
    • , Shylaja Arulkumar
    • , Hao Wan
    • , Malaya K Sahoo
    • , Janaki Abeynayake
    • , Jesse J Waggoner
    • , Clay Hopes
    • , Meijie Tang
    •  & Hongjie Dai
  • Brief Communication |

    Excess urinary cystine can lead to painful stone formation. There is no current effective treatment, but here Pankaj Kapahi, Marshall Stoller and colleagues have found that α-lipoic acid can prevent or even reverse formation of these stones in a mouse model.

    • Tiffany Zee
    • , Neelanjan Bose
    • , Jarcy Zee
    • , Jennifer N Beck
    • , See Yang
    • , Jaspreet Parihar
    • , Min Yang
    • , Sruthi Damodar
    • , David Hall
    • , Monique N O'Leary
    • , Arvind Ramanathan
    • , Roy R Gerona
    • , David W Killilea
    • , Thomas Chi
    • , Jay Tischfield
    • , Amrik Sahota
    • , Arnold Kahn
    • , Marshall L Stoller
    •  & Pankaj Kapahi
  • Brief Communication |

    New animal models of Zika virus (ZIKV) infection are imperative to accelerating efforts to treat or prevent disease in humans. Adams Waldorf et al. now report that ZIKV infection of a pregnant female pigtailed macaque caused brain lesions in the developing fetus, suggesting that this model may be useful for understanding ZIKV-associated congenital abnormalities in humans.

    • Kristina M Adams Waldorf
    • , Jennifer E Stencel-Baerenwald
    • , Raj P Kapur
    • , Colin Studholme
    • , Erica Boldenow
    • , Jay Vornhagen
    • , Audrey Baldessari
    • , Manjiri K Dighe
    • , Jeff Thiel
    • , Sean Merillat
    • , Blair Armistead
    • , Jennifer Tisoncik-Go
    • , Richard R Green
    • , Michael A Davis
    • , Elyse C Dewey
    • , Marian R Fairgrieve
    • , J Christopher Gatenby
    • , Todd Richards
    • , Gwenn A Garden
    • , Michael S Diamond
    • , Sandra E Juul
    • , Richard F Grant
    • , LaRene Kuller
    • , Dennis W W Shaw
    • , Jason Ogle
    • , G Michael Gough
    • , Wonsok Lee
    • , Chris English
    • , Robert F Hevner
    • , William B Dobyns
    • , Michael Gale Jr
    •  & Lakshmi Rajagopal
  • Brief Communication |

    By using combined positron emission and computed tomography (PET–CT), Esmail et al. show that some patients with latent tuberculosis have signs of subclinical, active disease in the lungs and a greater likelihood of progression, suggesting a spectrum of disease rather than discrete latent and active disease states.

    • Hanif Esmail
    • , Rachel P Lai
    • , Maia Lesosky
    • , Katalin A Wilkinson
    • , Christine M Graham
    • , Anna K Coussens
    • , Tolu Oni
    • , James M Warwick
    • , Qonita Said-Hartley
    • , Coenraad F Koegelenberg
    • , Gerhard Walzl
    • , JoAnne L Flynn
    • , Douglas B Young
    • , Clifton E Barry III
    • , Anne O'Garra
    •  & Robert J Wilkinson
  • Brief Communication |

    CRISPR–Cas9-mediated insertion of a naturally occurring benign mutation in blood cell progenitors from patients with sickle cell disease increases fetal hemoglobin expression to levels sufficient to ameliorate the pathological morphology observed in erythrocytes differentiated from these cells.

    • Elizabeth A Traxler
    • , Yu Yao
    • , Yong-Dong Wang
    • , Kaitly J Woodard
    • , Ryo Kurita
    • , Yukio Nakamura
    • , Jim R Hughes
    • , Ross C Hardison
    • , Gerd A Blobel
    • , Chunliang Li
    •  & Mitchell J Weiss
  • Brief Communication |

    Low-dose IL-2 treatment alters the abundance of regulatory T cells, IL-17-producing T cells and follicular helper T cells, but not of T helper type 1 and 2 cells, in patients with SLE.

    • Jing He
    • , Xia Zhang
    • , Yunbo Wei
    • , Xiaolin Sun
    • , Yaping Chen
    • , Jun Deng
    • , Yuebo Jin
    • , Yuzhou Gan
    • , Xin Hu
    • , Rulin Jia
    • , Chuanhui Xu
    • , Zhaohua Hou
    • , Yew Ann Leong
    • , Lei Zhu
    • , Jinhong Feng
    • , Yuan An
    • , Yuan Jia
    • , Chun Li
    • , Xu Liu
    • , Hua Ye
    • , Limin Ren
    • , Ru Li
    • , Haihong Yao
    • , Yuhui Li
    • , Shi Chen
    • , Xuewu Zhang
    • , Yin Su
    • , Jianping Guo
    • , Nan Shen
    • , Eric F Morand
    • , Di Yu
    •  & Zhanguo Li
  • Brief Communication |

    In mouse models of patient-derived breast cancer brain metastases, combined inhibition of PI3K and mTOR resulted in regression, and therapeutic response was correlated with a reduction in 4EBP1 phosphorylation.

    • Jing Ni
    • , Shakti H Ramkissoon
    • , Shaozhen Xie
    • , Shom Goel
    • , Daniel G Stover
    • , Hanbing Guo
    • , Victor Luu
    • , Eugenio Marco
    • , Lori A Ramkissoon
    • , Yun Jee Kang
    • , Marika Hayashi
    • , Quang-De Nguyen
    • , Azra H Ligon
    • , Rose Du
    • , Elizabeth B Claus
    • , Brian M Alexander
    • , Guo-Cheng Yuan
    • , Zhigang C Wang
    • , J Dirk Iglehart
    • , Ian E Krop
    • , Thomas M Roberts
    • , Eric P Winer
    • , Nancy U Lin
    • , Keith L Ligon
    •  & Jean J Zhao
  • Brief Communication |

    The microbiota of cesarean-born infants can be partially restored to that of vaginally born infants immediately after birth.

    • Maria G Dominguez-Bello
    • , Kassandra M De Jesus-Laboy
    • , Nan Shen
    • , Laura M Cox
    • , Amnon Amir
    • , Antonio Gonzalez
    • , Nicholas A Bokulich
    • , Se Jin Song
    • , Marina Hoashi
    • , Juana I Rivera-Vinas
    • , Keimari Mendez
    • , Rob Knight
    •  & Jose C Clemente
  • Brief Communication |

    An intracortical neural prosthetic system developed in animal studies is translated for clinical use in humans with paralysis. Neural control of computer cursor movements achieved with this system represent the highest performance reported to date.

    • Vikash Gilja
    • , Chethan Pandarinath
    • , Christine H Blabe
    • , Paul Nuyujukian
    • , John D Simeral
    • , Anish A Sarma
    • , Brittany L Sorice
    • , János A Perge
    • , Beata Jarosiewicz
    • , Leigh R Hochberg
    • , Krishna V Shenoy
    •  & Jaimie M Henderson
  • Brief Communication |

    Morgane Rolland and colleagues report that in HIV infection, a higher diversity of infecting founder viruses is associated with markers of poorer clinical outcome.

    • Holly Janes
    • , Joshua T Herbeck
    • , Sodsai Tovanabutra
    • , Rasmi Thomas
    • , Nicole Frahm
    • , Ann Duerr
    • , John Hural
    • , Lawrence Corey
    • , Steve G Self
    • , Susan P Buchbinder
    • , M Juliana McElrath
    • , Robert J O'Connell
    • , Robert M Paris
    • , Supachai Rerks-Ngarm
    • , Sorachai Nitayaphan
    • , Punnee Pitisuttihum
    • , Jaranit Kaewkungwal
    • , Merlin L Robb
    • , Nelson L Michael
    • , James I Mullins
    • , Jerome H Kim
    • , Peter B Gilbert
    •  & Morgane Rolland
  • Brief Communication |

    A small clinical trial shows that short-term cold acclimation to moderately-cold temperature improves the glucose homeostasis of individuals with type 2 diabetes, without an appreciable activation of their brown adipose tissue.

    • Mark J W Hanssen
    • , Joris Hoeks
    • , Boudewijn Brans
    • , Anouk A J J van der Lans
    • , Gert Schaart
    • , José J van den Driessche
    • , Johanna A Jörgensen
    • , Mark V Boekschoten
    • , Matthijs K C Hesselink
    • , Bas Havekes
    • , Sander Kersten
    • , Felix M Mottaghy
    • , Wouter D van Marken Lichtenbelt
    •  & Patrick Schrauwen
  • Brief Communication |

    The study describes the use of integrative approaches to search for candidate therapeutic targets for DIPG, and the identification of an HDAC inhibitor as a potential treatment strategy

    • Catherine S Grasso
    • , Yujie Tang
    • , Nathalene Truffaux
    • , Noah E Berlow
    • , Lining Liu
    • , Marie-Anne Debily
    • , Michael J Quist
    • , Lara E Davis
    • , Elaine C Huang
    • , Pamelyn J Woo
    • , Anitha Ponnuswami
    • , Spenser Chen
    • , Tessa B Johung
    • , Wenchao Sun
    • , Mari Kogiso
    • , Yuchen Du
    • , Lin Qi
    • , Yulun Huang
    • , Marianne Hütt-Cabezas
    • , Katherine E Warren
    • , Ludivine Le Dret
    • , Paul S Meltzer
    • , Hua Mao
    • , Martha Quezado
    • , Dannis G van Vuurden
    • , Jinu Abraham
    • , Maryam Fouladi
    • , Matthew N Svalina
    • , Nicholas Wang
    • , Cynthia Hawkins
    • , Javad Nazarian
    • , Marta M Alonso
    • , Eric H Raabe
    • , Esther Hulleman
    • , Paul T Spellman
    • , Xiao-Nan Li
    • , Charles Keller
    • , Ranadip Pal
    • , Jacques Grill
    •  & Michelle Monje
  • Brief Communication |

    A mutation conferring resistance to novel irreversible EGFR inhibitors is identified in cell-free plasma DNA from lung cancer patients.

    • Kenneth S Thress
    • , Cloud P Paweletz
    • , Enriqueta Felip
    • , Byoung Chul Cho
    • , Daniel Stetson
    • , Brian Dougherty
    • , Zhongwu Lai
    • , Aleksandra Markovets
    • , Ana Vivancos
    • , Yanan Kuang
    • , Dalia Ercan
    • , Sarah E Matthews
    • , Mireille Cantarini
    • , J Carl Barrett
    • , Pasi A Jänne
    •  & Geoffrey R Oxnard
  • Brief Communication |

    Goel et al. report that RIFINs mediate rosetting of Plasmodium falciparum infected erythrocytes in vitro, a phenotype that is associated with severe disease in humans

    • Suchi Goel
    • , Mia Palmkvist
    • , Kirsten Moll
    • , Nicolas Joannin
    • , Patricia Lara
    • , Reetesh R Akhouri
    • , Nasim Moradi
    • , Karin Öjemalm
    • , Mattias Westman
    • , Davide Angeletti
    • , Hanna Kjellin
    • , Janne Lehtiö
    • , Ola Blixt
    • , Lars Ideström
    • , Carl G Gahmberg
    • , Jill R Storry
    • , Annika K Hult
    • , Martin L Olsson
    • , Gunnar von Heijne
    • , IngMarie Nilsson
    •  & Mats Wahlgren
  • Brief Communication |

    Aftab Ansari and his colleagues show that antibody-mediated masking of α4β7 integrin impedes intravaginal transmission of simian immunodeficiency virus in macaques.

    • Siddappa N Byrareddy
    • , Brianne Kallam
    • , James Arthos
    • , Claudia Cicala
    • , Fatima Nawaz
    • , Joseph Hiatt
    • , Ellen N Kersh
    • , Janet M McNicholl
    • , Debra Hanson
    • , Keith A Reimann
    • , Markus Brameier
    • , Lutz Walter
    • , Kenneth Rogers
    • , Ann E Mayne
    • , Paul Dunbar
    • , Tara Villinger
    • , Dawn Little
    • , Tristram G Parslow
    • , Philip J Santangelo
    • , Francois Villinger
    • , Anthony S Fauci
    •  & Aftab A Ansari
  • Brief Communication |

    Hashizume et al. report a new therapeutic strategy for treating pediatric gliomas with mutations in the H3F3A gene by inhibiting histone demethylation.

    • Rintaro Hashizume
    • , Noemi Andor
    • , Yuichiro Ihara
    • , Robin Lerner
    • , Haiyun Gan
    • , Xiaoyue Chen
    • , Dong Fang
    • , Xi Huang
    • , Maxwell W Tom
    • , Vy Ngo
    • , David Solomon
    • , Sabine Mueller
    • , Pamela L Paris
    • , Zhiguo Zhang
    • , Claudia Petritsch
    • , Nalin Gupta
    • , Todd A Waldman
    •  & C David James
  • Brief Communication |

    A chimpanzee adenovirus-based vaccination approach elicits acute and long-term protection against ebolavirus challenge in nonhuman primates.

    • Daphne A Stanley
    • , Anna N Honko
    • , Clement Asiedu
    • , John C Trefry
    • , Annie W Lau-Kilby
    • , Joshua C Johnson
    • , Lisa Hensley
    • , Virginia Ammendola
    • , Adele Abbate
    • , Fabiana Grazioli
    • , Kathryn E Foulds
    • , Cheng Cheng
    • , Lingshu Wang
    • , Mitzi M Donaldson
    • , Stefano Colloca
    • , Antonella Folgori
    • , Mario Roederer
    • , Gary J Nabel
    • , John Mascola
    • , Alfredo Nicosia
    • , Riccardo Cortese
    • , Richard A Koup
    •  & Nancy J Sullivan
  • Brief Communication |

    Tau pathology is identified in brains of individuals with Huntington's disease (HD), and reduction of tau expression can ameliorate disease in HD model mice.

    • Marta Fernández-Nogales
    • , Jorge R Cabrera
    • , María Santos-Galindo
    • , Jeroen J M Hoozemans
    • , Isidro Ferrer
    • , Annemieke J M Rozemuller
    • , Félix Hernández
    • , Jesús Avila
    •  & José J Lucas
  • Brief Communication |

    Pancreatic adenosquamous carcinomas are rare but highly aggressive tumors, and our understanding of them is limited. In a new study, Miles Wilkinson and colleagues now report that they have identified somatic mutations in an RNA helicase encoded by UPF1 in some pancreatic adenosquamous carcinomas. UPF1 is required for nonsense-mediated RNA decay, suggesting that the aberrant accumulation of altered RNA transcripts may contribute to the malignant phenotype of these tumors and could be a target of diagnostic and therapeutic approaches for this tumor type.

    • Chen Liu
    • , Rachid Karam
    • , YingQi Zhou
    • , Fang Su
    • , Yuan Ji
    • , Gang Li
    • , GuoTong Xu
    • , LiXia Lu
    • , ChongRen Wang
    • , MeiYi Song
    • , JingPing Zhu
    • , YiRan Wang
    • , YiFan Zhao
    • , Wai Chin Foo
    • , MingXin Zuo
    • , Mark A Valasek
    • , Milind Javle
    • , Miles F Wilkinson
    •  & YanJun Lu
  • Brief Communication |

    Mutations inactivating ARID1A, a subunit of the chromatin remodeling SWI/SNF complex, have been identified in some human cancers. This study reveals that cancer cells with mutated ARID1A are dependent on the residual activity of the complex for proliferation and that even if concomitant alterations in the ARID1A homolog ARID1B can occur, loss of ARID1B activity confers a specific vulnerability to ARID1A-mutant cells that may in the future be explored for targeting purposes.

    • Katherine C Helming
    • , Xiaofeng Wang
    • , Boris G Wilson
    • , Francisca Vazquez
    • , Jeffrey R Haswell
    • , Haley E Manchester
    • , Youngha Kim
    • , Gregory V Kryukov
    • , Mahmoud Ghandi
    • , Andrew J Aguirre
    • , Zainab Jagani
    • , Zhong Wang
    • , Levi A Garraway
    • , William C Hahn
    •  & Charles W M Roberts
  • Brief Communication |

    Understanding how and where HIV-1 infection persists in the body is crucial for efforts to eradicate the viral reservoir. Now Mathias Lichterfeld and his colleagues report that HIV-1 can infect CD4+ T memory stem cells (TSCM cells) and that infected TSCM cells constitute a stable component of the viral reservoir in individuals treated with antiretroviral therapy.

    • Maria J Buzon
    • , Hong Sun
    • , Chun Li
    • , Amy Shaw
    • , Katherine Seiss
    • , Zhengyu Ouyang
    • , Enrique Martin-Gayo
    • , Jin Leng
    • , Timothy J Henrich
    • , Jonathan Z Li
    • , Florencia Pereyra
    • , Ryan Zurakowski
    • , Bruce D Walker
    • , Eric S Rosenberg
    • , Xu G Yu
    •  & Mathias Lichterfeld