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Showing 1–21 of 21 results
Advanced filters: Author: Aravind Asokan Clear advanced filters

  • Stable and cell-specific transgene expression can be achieved through in vivo site-specific integration of large DNA payloads using a two-vector system of enveloped delivery vehicles and adeno-associated viruses.

    • William A. Nyberg
    • Pierre-Louis Bernard
    • Justin Eyquem
    ResearchOpen Access
    Nature
    P: 1-10

  • The ability to edit large stretches of mRNA transcripts remains a significant challenge. Here, the authors demonstrate that CRISPR-Cas13 systems can be repurposed to assist trans-splicing of exogenous RNA fragments into an endogenous pre-mRNA transcript, a method termed CRISPR Assisted mRNA Fragment Trans-splicing (CRAFT).

    • David N. Fiflis
    • Nicolas A. Rey
    • Aravind Asokan
    ResearchOpen Access
    Nature Communications
    Volume: 15, P: 1-14

  • Gene therapy vectors based on adeno-associated virus tend to accumulate in the liver, limiting their utility for targeting other organs. Using site-directed mutagenesis of the capsid surface, Asokan et al. generate a variant that efficiently transduces a wide range of muscle groups while avoiding the liver.

    • Aravind Asokan
    • Julia C Conway
    • R Jude Samulski
    Research
    Nature Biotechnology
    Volume: 28, P: 79-82

  • Gonzalez et al. use a viral evolutionary approach to generate cross-species compatible AAV (ccAAVs) vectors. They describe a highly potent new variant, AAV.cc47, with enhanced transduction efficiency over AAV serotype 9 and show its efficacy in different mouse models, pigs and non-human primates.

    • Trevor J. Gonzalez
    • Katherine E. Simon
    • Aravind Asokan
    ResearchOpen Access
    Nature Communications
    Volume: 13, P: 1-17

  • Variants of adeno-associated viruses displaying robust and widespread transduction in murine, porcine and non-human-primate kidneys, and in human kidney organoids, can be obtained via the cross-species cycling of capsid libraries in the different kidney models.

    • Alan Rosales
    • Leo O. Blondel
    • Aravind Asokan
    ResearchOpen Access
    Nature Biomedical Engineering
    Volume: 9, P: 1086-1100

  • Adeno-associated viruses can be secreted in a pre-lytic manner as free or extracellular vesicle (EV)-associated particles. Here, Elmore et al. show that the recently identified membrane-associated accessory protein (MAAP) functions as an AAV egress factor via association to EVs.

    • Zachary C. Elmore
    • L. Patrick Havlik
    • Aravind Asokan
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-13

  • Hematopoietic stem and progenitor cells have been engineered using gold nanoformulations conjugated with CRISPR capable of targeting two distinct genomic loci of therapeutic interest, with potential engraftment in humanized mouse models.

    • Aravind Asokan
    News & Views
    Nature Materials
    Volume: 18, P: 1038-1039

  • Formation of biomolecular condensates composed of proteins and RNA facilitates the regulation of gene expression by modulating translation or facilitating RNA processing. Now, synthetic ribonucleoprotein granules created with engineered intrinsically disordered proteins selectively sequester mRNA and enhance protein translation in cells. These highly liquid-like condensates exchange biomolecules across the cell and facilitate target mRNA and ribosome partitioning.

    • Daniel Mark Shapiro
    • Sonal Deshpande
    • Ashutosh Chilkoti
    Research
    Nature Chemistry
    Volume: 17, P: 448-456

  • All natural AAV serotypes transduce murine hepatocytes more efficiently than their human counterparts in human liver chimeric mouse models. Here the authors developed a novel humanized mouse were human transduction of AAV can be studied.

    • Mercedes Barzi
    • Tong Chen
    • Karl-Dimiter Bissig
    ResearchOpen Access
    Nature Communications
    Volume: 15, P: 1-10

  • This protocol provides structure-guided capsid library approaches to evolve new adeno-associated viral vectors for enhanced CNS gene delivery with altered tissue tropism, higher transduction efficiency and the ability to evade pre-existing humoral immunity.

    • Trevor J. Gonzalez
    • Aaron Mitchell-Dick
    • Aravind Asokan
    Protocols
    Nature Protocols
    Volume: 18, P: 3413-3459

  • This Perspective discusses how the Somatic Cell Genome Editing Consortium aims to accelerate the implementation of safe and effective genome-editing therapies in the clinic.

    • Krishanu Saha
    • Erik J. Sontheimer
    • Jiangbing Zhou
    ReviewsOpen Access
    Nature
    Volume: 592, P: 195-204

  • Zebrafish can regenerate after paralyzing spine injuries and regain locomotor ability, unlike mammals. Here authors show that the neurogenic factor Hb-egf promotes spinal cord regeneration in zebrafish and is regulated by an enhancer that can similarly direct expression in the pro-regenerative setting of neonatal mice.

    • Valentina Cigliola
    • Adam Shoffner
    • Kenneth D. Poss
    ResearchOpen Access
    Nature Communications
    Volume: 14, P: 1-18

  • Hepatitis B virus (HBV) has a limited host range and current animal models can only recapitulate certain aspects of HBV replication. Here, the authors show that expression of the HBV receptor NTCP in macaques supports HBV replication in vivo, suggesting this as animal model for future HBV studies.

    • Benjamin J. Burwitz
    • Jochen M. Wettengel
    • Jonah B. Sacha
    ResearchOpen Access
    Nature Communications
    Volume: 8, P: 1-10

  • Directed evolution of adeno-associated virus (AAV) yields mutant vectors that can evade neutralizing antibodies.

    • Aravind Asokan
    • R Jude Samulski
    News & Views
    Nature Biotechnology
    Volume: 24, P: 158-160

  • In the mdx mouse model of Duchenne muscular dystrophy, single intravenous administration of AAV-CRISPR–Cas9 vectors provides efficient genome editing and restoration of dystrophin expression lasting for one year.

    • Christopher E. Nelson
    • Yaoying Wu
    • Charles A. Gersbach
    Research
    Nature Medicine
    Volume: 25, P: 427-432