Focus

Therapeutics

Welcome to the Nature Communications Editors’ Highlights webpage on therapeutics. Each month our editors select a small number of Articles recently published in Nature Communications that they believe are particularly interesting or important.

The aim is to provide a snapshot of some of the most exciting work published in the area of therapeutics at Nature Communications. Each editor handles a different area of this research, as described below:

Ross Cloney handles therapeutic biotechnology, including manuscripts that involve novel vectors, nanoparticles, synthetic biology and genome engineering approaches. 

Francesco Conti handles manuscripts on muscle and bone biology, gene therapy, therapeutics, preclinical and clinical studies.

Aishwarya Sundaram handles manuscripts on cancer drug discovery, cancer therapy, nanotherapy, metastasis, and tumor models. 

Ross Cloney

  • Nature Communications | Article | open

    Viral gene therapy can be limited by the efficacy of virion sequestration, immune responses and the silencing of genetic payloads. Here the authors engineer an advenovirus protein coat which shields the virion from the immune system while targeting cancer cells.

    • Markus Schmid
    • , Patrick Ernst
    • , Annemarie Honegger
    • , Maarit Suomalainen
    • , Martina Zimmermann
    • , Lukas Braun
    • , Sarah Stauffer
    • , Cristian Thom
    • , Birgit Dreier
    • , Matthias Eibauer
    • , Anja Kipar
    • , Viola Vogel
    • , Urs F. Greber
    • , Ohad Medalia
    •  &  Andreas Plückthun
  • Nature Communications | Article | open

    Exosomes function as intercellular information transmitters and are candidates for delivery of therapeutic agents. Here the authors present EXOtic, a synthetic biology device for in-situ production of designer exosomes and demonstrate in vivo application in models of Parkinson's disease.

    • Ryosuke Kojima
    • , Daniel Bojar
    • , Giorgio Rizzi
    • , Ghislaine Charpin-El Hamri
    • , Marie Daoud El-Baba
    • , Pratik Saxena
    • , Simon Ausländer
    • , Kelly R. Tan
    •  &  Martin Fussenegger
  • Nature Communications | Article | open

    The restoration of light response using retinal prosthesis could be a way to restore vision following retinal degenerative disease. Here the authors develop gold-titania nanowire arrays that restore visual response in blind mice.

    • Jing Tang
    • , Nan Qin
    • , Yan Chong
    • , Yupu Diao
    • , Yiliguma
    • , Zhexuan Wang
    • , Tian Xue
    • , Min Jiang
    • , Jiayi Zhang
    •  &  Gengfeng Zheng
  • Nature Communications | Article | open

    Droplet-based single-cell RNA-seq is a powerful tool for cellular heterogeneity profiling in disease but is limited by instrumentation required. Here the authors develop a 3D printed microfluidic platform for massive parallel sequencing of rheumatoid arthritis tissues.

    • William Stephenson
    • , Laura T. Donlin
    • , Andrew Butler
    • , Cristina Rozo
    • , Bernadette Bracken
    • , Ali Rashidfarrokhi
    • , Susan M. Goodman
    • , Lionel B. Ivashkiv
    • , Vivian P. Bykerk
    • , Dana E. Orange
    • , Robert B. Darnell
    • , Harold P. Swerdlow
    •  &  Rahul Satija
  • Nature Communications | Article | open

    The treatment of inflammatory arthritis by local delivery of therapeutics is limited by short half-lives of drugs. Here the authors demonstrate a hydrogel platform that titrates drug release to arthritis activity.

    • Nitin Joshi
    • , Jing Yan
    • , Seth Levy
    • , Sachin Bhagchandani
    • , Kai V. Slaughter
    • , Nicholas E. Sherman
    • , Julian Amirault
    • , Yufeng Wang
    • , Logan Riegel
    • , Xueyin He
    • , Tan Shi Rui
    • , Michael Valic
    • , Praveen K. Vemula
    • , Oscar R. Miranda
    • , Oren Levy
    • , Ellen M. Gravallese
    • , Antonios O. Aliprantis
    • , Joerg Ermann
    •  &  Jeffrey M. Karp
  • Nature Communications | Article | open

    Self-assembling proteins have important applications in industrial technologies, but it is difficult to predict how mutations affect assembly. Here the authors present SyMAPS, coupling comprehensive codon mutagenesis with high-throughput sequencing, and apply it to bacteriophage capsid protein.

    • Emily C. Hartman
    • , Christopher M. Jakobson
    • , Andrew H. Favor
    • , Marco J. Lobba
    • , Ester Álvarez-Benedicto
    • , Matthew B. Francis
    •  &  Danielle Tullman-Ercek

Francesco Conti

  • Nature Communications | Article | open

    Fibrodysplasia ossificans progressiva is a severe disorder characterized by heterotopic ossification, and is caused by mutations in ACVR1. Here, the authors show that expression of mutant ACVR1 in fibro/adipogenic progenitors recapitulates disease progression, and that this can be halted by systemic inhibition of activin A in mice.

    • John B. Lees-Shepard
    • , Masakazu Yamamoto
    • , Arpita A. Biswas
    • , Sean J. Stoessel
    • , Sarah-Anne E. Nicholas
    • , Cathy A. Cogswell
    • , Parvathi M. Devarakonda
    • , Michael J. Schneider Jr.
    • , Samantha M. Cummins
    • , Nicholas P. Legendre
    • , Shoko Yamamoto
    • , Vesa Kaartinen
    • , Jeffrey W. Hunter
    •  &  David J. Goldhamer
  • Nature Communications | Article | open

    Heterotopic ossification (HO) is a painful disease of unknown etiology characterized by extraskeletal bone formation after injury. Here the authors show that TGF-β is increased in HO lesions, where it promotes the early stages of HO pathology, and demonstrate that TGF-β inhibition ameliorates HO in mice.

    • Xiao Wang
    • , Fengfeng Li
    • , Liang Xie
    • , Janet Crane
    • , Gehua Zhen
    • , Yuji Mishina
    • , Ruoxian Deng
    • , Bo Gao
    • , Hao Chen
    • , Shen Liu
    • , Ping Yang
    • , Manman Gao
    • , Manli Tu
    • , Yiguo Wang
    • , Mei Wan
    • , Cunyi Fan
    •  &  Xu Cao
  • Nature Communications | Article | open

    Satellite cells can differentiate both into myocytes and brown adipocytes. Here, the authors show that the histone demethylase Lsd1 prevents adipogenic differentiation of satellite cells by repressing expression of Glis1, and that its ablation changes satellite cell fate towards brown adipocytes and delays muscle regeneration in mice.

    • Milica Tosic
    • , Anita Allen
    • , Dominica Willmann
    • , Christoph Lepper
    • , Johnny Kim
    • , Delphine Duteil
    •  &  Roland Schüle
  • Nature Communications | Article | open

    Short-chain fatty acids (SCFA) are a main class of metabolites derived from fermentation of dietary fibre in the intestine. Here, the authors show that dietary administration of SCFA is associated with inhibition of osteoclast differentiation, increased bone mass, and reduced pathological bone loss in mice.

    • Sébastien Lucas
    • , Yasunori Omata
    • , Jörg Hofmann
    • , Martin Böttcher
    • , Aida Iljazovic
    • , Kerstin Sarter
    • , Olivia Albrecht
    • , Oscar Schulz
    • , Brenda Krishnacoumar
    • , Gerhard Krönke
    • , Martin Herrmann
    • , Dimitrios Mougiakakos
    • , Till Strowig
    • , Georg Schett
    •  &  Mario M. Zaiss
  • Nature Communications | Article | open

    Progressive multiple sclerosis is an inflammatory and degenerative disease of the central nervous system, for which effective treatment is lacking. The authors carry out a screen to identify orally available generic medications, and show that the antidepressant clomipramine reduces pathology in mouse models.

    • Simon Faissner
    • , Manoj Mishra
    • , Deepak K. Kaushik
    • , Jianxiong Wang
    • , Yan Fan
    • , Claudia Silva
    • , Gail Rauw
    • , Luanne Metz
    • , Marcus Koch
    •  &  V. Wee Yong
  • Nature Communications | Article | open

    The generation of functional skeletal muscle tissue from human pluripotent stem cells has not been reported. Here, the authors describe engineering of contractile skeletal muscle bundles in culture, which become vascularized and maintain functionality when transplanted into mice.

    • Lingjun Rao
    • , Ying Qian
    • , Alastair Khodabukus
    • , Thomas Ribar
    •  &  Nenad Bursac

Aishwarya Sundaram

  • Nature Communications | Article | open

    Imaging in the second near-infrared window has attracted attention due to superior penetration depth and low signal interference. Here, the authors describe a new organic nano fluorophore with high quantum yield and demonstrate its use for in vivo imaging.

    • Hao Wan
    • , Jingying Yue
    • , Shoujun Zhu
    • , Takaaki Uno
    • , Xiaodong Zhang
    • , Qinglai Yang
    • , Kuai Yu
    • , Guosong Hong
    • , Junying Wang
    • , Lulin Li
    • , Zhuoran Ma
    • , Hongpeng Gao
    • , Yeteng Zhong
    • , Jessica Su
    • , Alexander L. Antaris
    • , Yan Xia
    • , Jian Luo
    • , Yongye Liang
    •  &  Hongjie Dai
  • Nature Communications | Article | open

    Activation of the tumor suppressor p53 is a promising approach in cancer therapy. Here, the authors discover a series of small molecule dihydroorotate dehydrogenase (DHODH) inhibitors that increase p53 synthesis and reduce tumor growth in synergy with the common mdm2 inhibitor nutlin3.

    • Marcus J. G. W. Ladds
    • , Ingeborg M. M. van Leeuwen
    • , Catherine J. Drummond
    • , Su Chu
    • , Alan R. Healy
    • , Gergana Popova
    • , Andrés Pastor Fernández
    • , Tanzina Mollick
    • , Suhas Darekar
    • , Saikiran K. Sedimbi
    • , Marta Nekulova
    • , Marijke C. C. Sachweh
    • , Johanna Campbell
    • , Maureen Higgins
    • , Chloe Tuck
    • , Mihaela Popa
    • , Mireia Mayoral Safont
    • , Pascal Gelebart
    • , Zinayida Fandalyuk
    • , Alastair M. Thompson
    • , Richard Svensson
    • , Anna-Lena Gustavsson
    • , Lars Johansson
    • , Katarina Färnegårdh
    • , Ulrika Yngve
    • , Aljona Saleh
    • , Martin Haraldsson
    • , Agathe C. A. D’Hollander
    • , Marcela Franco
    • , Yan Zhao
    • , Maria Håkansson
    • , Björn Walse
    • , Karin Larsson
    • , Emma M. Peat
    • , Vicent Pelechano
    • , John Lunec
    • , Borivoj Vojtesek
    • , Mar Carmena
    • , William C. Earnshaw
    • , Anna R. McCarthy
    • , Nicholas J. Westwood
    • , Marie Arsenian-Henriksson
    • , David P. Lane
    • , Ravi Bhatia
    • , Emmet McCormack
    •  &  Sonia Laín
  • Nature Communications | Article | open

    Triple-negative breast cancer (TNBC) is one of the most aggressive and difficult to treat types of breast cancer. Here, a phase 2 clinical trial in TNBC patients reveals that the addition of lobaplatin to docetaxel and epirubicin regime improves pCR and ORR rates with tolerable side-effects.

    • Xiujuan Wu
    • , Peng Tang
    • , Shifei Li
    • , Shushu Wang
    • , Yueyang Liang
    • , Ling Zhong
    • , Lin Ren
    • , Ting Zhang
    •  &  Yi Zhang
  • Nature Communications | Article | open

    Photothermal therapy (PTT) for cancer treatment is currently limited to local, accessible, tumors. Here the authors show that PTT combination with chemotherapy, by stimulating an immune response, is effective against distant tumors and establishes immune memory, thus providing a strategy to target metastatic disease.

    • Jutaek Nam
    • , Sejin Son
    • , Lukasz J. Ochyl
    • , Rui Kuai
    • , Anna Schwendeman
    •  &  James J. Moon
  • Nature Communications | Article | open

    Tumor cells can fuel their metabolism with lactate. Here the authors show that inhibition of mitochondrial pyruvate carrier (MPC) blocks extracellular lactate uptake by promoting intracellular pyruvate accumulation and inhibits oxidative metabolism, ultimately resulting in cytotoxicity and radiosensitization.

    • Cyril Corbet
    • , Estelle Bastien
    • , Nihed Draoui
    • , Bastien Doix
    • , Lionel Mignion
    • , Bénédicte F. Jordan
    • , Arnaud Marchand
    • , Jean-Christophe Vanherck
    • , Patrick Chaltin
    • , Olivier Schakman
    • , Holger M. Becker
    • , Olivier Riant
    •  &  Olivier Feron
  • Nature Communications | Article | open

    Patients’ selection is particularly important in cancer treatment. Here the authors present a proof-of-principle methodology that could be potentially important in assisting therapeutic decisions in the treatment of breast cancer patients.

    • Valeriy Domenyuk
    • , Zoran Gatalica
    • , Radhika Santhanam
    • , Xixi Wei
    • , Adam Stark
    • , Patrick Kennedy
    • , Brandon Toussaint
    • , Symon Levenberg
    • , Jie Wang
    • , Nianqing Xiao
    • , Richard Greil
    • , Gabriel Rinnerthaler
    • , Simon P. Gampenrieder
    • , Amy B. Heimberger
    • , Donald A. Berry
    • , Anna Barker
    • , John Quackenbush
    • , John L. Marshall
    • , George Poste
    • , Jeffrey L. Vacirca
    • , Gregory A. Vidal
    • , Lee S. Schwartzberg
    • , David D. Halbert
    • , Andreas Voss
    • , Daniel Magee
    • , Mark R. Miglarese
    • , Michael Famulok
    • , Günter Mayer
    •  &  David Spetzler