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    • Size does matter: overcoming the adeno-associated virus packaging limit

    Metadata Updated: September 7, 2025

    Recombinant adeno-associated virus (rAAV) vectors mediate long-term gene transfer without any known toxicity. The primary limitation of rAAV has been the small size of the virion (20 nm), which only permits the packaging of 4.7 kilobases (kb) of exogenous DNA, including the promoter, the polyadenylation signal and any other enhancer elements that might be desired. Two recent reports (D Duan et al: Nat Med 2000, 6:595-598; Z Yan et al: Proc Natl Acad Sci USA 2000, 97:6716-6721) have exploited a unique feature of rAAV genomes, their ability to link together in doublets or strings, to bypass this size limitation. This technology could improve the chances for successful gene therapy of diseases like cystic fibrosis or Duchenne muscular dystrophy that lead to significant pulmonary morbidity.

    Access & Use Information

    Public: This dataset is intended for public access and use. License: No license information was provided. If this work was prepared by an officer or employee of the United States government as part of that person's official duties it is considered a U.S. Government Work.

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    Dates

    Metadata Created Date July 24, 2025
    Metadata Updated Date September 7, 2025

    Metadata Source

    Harvested from Healthdata.gov

    Other Data Resources

    Additional Metadata

    Resource Type Dataset
    Metadata Created Date July 24, 2025
    Metadata Updated Date September 7, 2025
    Publisher National Institutes of Health
    Maintainer
    NIH
    Identifier https://healthdata.gov/api/views/u264-ty9p
    Data First Published 2025-07-14
    Data Last Modified 2025-09-06
    Category NIH
    Public Access Level public
    Bureau Code 009:25
    Metadata Context https://project-open-data.cio.gov/v1.1/schema/catalog.jsonld
    Metadata Catalog ID https://healthdata.gov/data.json
    Schema Version https://project-open-data.cio.gov/v1.1/schema
    Catalog Describedby https://project-open-data.cio.gov/v1.1/schema/catalog.json
    Harvest Object Id aba56936-a2c5-436a-9af7-8f88514941e0
    Harvest Source Id 651e43b2-321c-4e4c-b86a-835cfc342cb0
    Harvest Source Title Healthdata.gov
    Homepage URL https://healthdata.gov/d/u264-ty9p
    Program Code 009:033
    Source Datajson Identifier True
    Source Hash 708ddf1e1bbda99f46b1e0a0683156e6e63b69a889c11d5a5405745d20e05e93
    Source Schema Version 1.1

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