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    • Taking stock of gene therapy for cystic fibrosis

    Metadata Updated: September 6, 2025

    The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal models in vivo has been followed by numerous clinical studies using both viral and non-viral vectors to transfer normal copies of the gene to the lungs and noses of CF patients. A wealth of data have emerged from these studies, reflecting enormous progress and also helping to focus and define key difficulties that remain unresolved. Gene therapy for CF remains the most promising possibility for curative rather than symptomatic therapy.

    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 6, 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 6, 2025
    Publisher National Institutes of Health
    Maintainer
    NIH
    Identifier https://healthdata.gov/api/views/3b35-df6i
    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 23201379-fea6-4758-a200-2c39d76cb2bd
    Harvest Source Id 651e43b2-321c-4e4c-b86a-835cfc342cb0
    Harvest Source Title Healthdata.gov
    Homepage URL https://healthdata.gov/d/3b35-df6i
    Program Code 009:033
    Source Datajson Identifier True
    Source Hash 6c421d44465e1683459556112502210302609dbd13641e2ba2760db72e56d1f7
    Source Schema Version 1.1

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