Gene delivery articles within Nature Communications

Featured

  • Article
    | Open Access

    Interfering RNA have a range of therapeutic and research based applications, issues with delivery have made systems that make siRNA in situ of interest. Here, the author report on the creation of a DNA hydrogel with improved stability and transcription efficiency over plasmid DNA.

    • Jaejung Song
    • , Minhyuk Lee
    •  & Nokyoung Park
  • Article
    | Open Access

    Immunogenicity of AAV vectors renders repeated AAV dosing ineffective. Here the authors show that coadministration of nanoparticle-encapsulated rapamycin overcomes AAV immunogenicity through Treg induction, enabling efficient AAV redosing in mice and nonhuman primates.

    • Amine Meliani
    • , Florence Boisgerault
    •  & Federico Mingozzi
  • Article
    | Open Access

    Activation of the NLRP3 inflammasome triggers the production of inflammatory cytokines. Here, the authors inactivate NLRP3 in macrophages using CRISPR/Cas9 encapsulated in nanoparticles, and show that administration in mice is effective in preventing septic shock and peritonitis, and in improving diabetes-associated inflammation and insulin resistance.

    • Congfei Xu
    • , Zidong Lu
    •  & Jun Wang
  • Article
    | Open Access

    Single-particle cryo-EM is a powerful method for macromolecular structure determination. Here the authors demonstrate that Ewald sphere curvature correction, sub-Angstrom pixilation and per-particle CTF refinement can improve map quality and resolution and present the 1.86 Å cryo-EM structure of an adeno-associated virus serotype 2 variant.

    • Yong Zi Tan
    • , Sriram Aiyer
    •  & Dmitry Lyumkis
  • Article
    | Open Access

    Classic monodirectional promoters are of limited use for multiple gene co-expression. Here the authors generate a library of 168 bidirectional promoters for the yeast K. phaffii (syn. P. pastoris) with diverse expression profiles to optimize metabolic pathway design.

    • Thomas Vogl
    • , Thomas Kickenweiz
    •  & Anton Glieder
  • Article
    | Open Access

    X-ray radiation has excellent tissue penetration depth, making it a useful trigger for deep tissue cancer therapy. Here, the authors design X-ray triggered drug/gene-loaded liposomes by embedding photosensitizers and gold nanoparticles in the liposome bilayer, and demonstrate their efficacy in cancer and gene therapy.

    • Wei Deng
    • , Wenjie Chen
    •  & Ewa M. Goldys
  • Article
    | Open Access

    In vivo reprogramming of somatic cells is hampered by the need for vectors to express the OKSM factors in selected organs. Here the authors report new AAV-based vectors capable of in vivo reprogramming at low doses.

    • Elena Senís
    • , Lluc Mosteiro
    •  & Dirk Grimm
  • Article
    | Open Access

    Delivery of therapeutic proteins to patients may be costly to administer, and often requires repeated injections. Here, the authors use antigen-specific T cells expressing erythropoietin, and show that following transfer in mice, cell expansion and erythropoietin production can be boosted by vaccination, raising the haematocrit.

    • Richard T. O’Neil
    • , Sunandan Saha
    •  & Matthew H. Wilson
  • Article
    | Open Access

    Current widely used viral and electroporation methods for creating therapeutic cell-based products are complex and expensive. Here, the authors develop targeted mRNA nanocarriers that can transiently program gene expression by simply mixing them with cells, to improve their therapeutic potential.

    • H. F. Moffett
    • , M. E. Coon
    •  & M. T. Stephan
  • Article
    | Open Access

    Proliferation of synoviocytes contributes to joint damage in rheumatoid arthritis. Here the authors show that targeting of these cells by a vector, consisting of a baculovirus conjugated to an adenovirus carrying the pro-apoptotic gene PUMA, has therapeutic efficacy in a rat arthritis model.

    • Saw-See Hong
    • , Hubert Marotte
    •  & Pierre Miossec
  • Article
    | Open Access

    Single-cell characterization and perturbation of neurons is critical for revealing the structure-function relationship of brain cells. Here the authors develop a robot that performs single-cell electroporation and extracellular electrophysiology and can be used for performingin vivosingle-cell experiments in deep brain tissues optically difficult to access.

    • Lu Li
    • , Benjamin Ouellette
    •  & Hongkui Zeng
  • Article
    | Open Access

    The maximum titre of therapeutic viral vectors can be adversely affected by the encoded transgene. Here the authors repress transgene expression in producing cells by employing the tryptophan RNA-binding attenuation protein and show that it improves titre of RNA- and DNA-based viral vectors expressing toxic transgenes.

    • H. E. Maunder
    • , J. Wright
    •  & D. C. Farley
  • Article
    | Open Access

    The anti-diabetic drug Metformin also possesses anti-tumour activity. Here, the authors synthesize polymeric Metformin-based nanoparticles that still exert intrinsic biological activity through AMPK and mTOR regulation and can systematically deliver VEGF siRNA, significantly reducing lung cancer growth in mice.

    • Yi Zhao
    • , Wei Wang
    •  & Leaf Huang
  • Article
    | Open Access

    Current viral gene delivery systems are limited in the amount of foreign DNA they can deliver to cells. Here the authors develop MultiPrime, a baculovirus-based vector system capable of multigene delivery into a wide variety of cells, and use Multiprime for genome engineering by CRISPR/Cas9.

    • Maysam Mansouri
    • , Itxaso Bellon-Echeverria
    •  & Philipp Berger
  • Article
    | Open Access

    Algae hold great promise for biofuel and chemical production but their use as model systems is hampered by the absence of suitable genetic tools. Here Karas et al. present a nuclear episomal vector for diatoms that is maintained in the absence of antibiotics, and a plasmid delivery method via conjugation with E. coli.

    • Bogumil J. Karas
    • , Rachel E. Diner
    •  & Philip D. Weyman
  • Article |

    The clinical use of adeno-associated virus vectors (AAVs) has been limited by the lack of transduction specificity. Here the authors show that receptor-targeted, affinity-tagged, and purified AVVs reach tumours in mouse models with high selectivity and efficiency, outperforming therapeutic antibodies.

    • Robert C. Münch
    • , Anke Muth
    •  & Christian J. Buchholz
  • Article |

    The incorporation of foreign objects into cells can be used in various avenues of biological research, although crossing the cell membrane can be challenging. Here, the authors use a diamond nanoneedle array for enhanced delivery of various particles into cells, including neurons.

    • Ying Wang
    • , Yang Yang
    •  & Peng Shi
  • Article |

    Robust and reliable structure–function relationships are valuable for the development of potent drug delivery systems. Here, the authors use a library of lipid-like materials to predict in vivosiRNA delivery efficacy without any biological testing.

    • Kathryn A. Whitehead
    • , J. Robert Dorkin
    •  & Daniel G. Anderson
  • Article
    | Open Access

    In uteroelectroporation allows the labelling of specific populations of neurons in the developing mouse brain. The authors of this paper present a simple modification of this method that increases labelling efficiency and allows, for the first time, transfection of Purkinje cells in the rat cerebellum.

    • Marco dal Maschio
    • , Diego Ghezzi
    •  & Laura Cancedda
  • Article |

    Fluorescent proteins are widely used in molecular biology to visualize protein expression and localization. Here, Krizet al. describe an efficient and flexible modular plasmid-based eukaryotic expression cloning strategy for the homogeneous expression of several fluorescent proteins in one cell.

    • Andrijana Kriz
    • , Katharina Schmid
    •  & Philipp Berger