Gene therapy progress and prospects: targeted gene repair View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2005-04

AUTHORS

H Parekh-Olmedo, L Ferrara, E Brachman, E B Kmiec

ABSTRACT

The capacity to correct a mutant gene within the context of the chromosome holds great promise as a therapy for inherited disorders but fulfilling this promise has proven to be challenging. However, steady progress is being made and the development of gene repair as a viable and robust approach is underway. Here, we present some of the recent advances that are helping to shape our thinking about the feasibility and the limitations of this technique. For the most part, these advances center on understanding the regulation of the reaction and validating its application in animal models. More... »

PAGES

3302511

References to SciGraph publications

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  • Journal

    TITLE

    Gene Therapy

    ISSUE

    8

    VOLUME

    12

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  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/sj.gt.3302511

    DOI

    http://dx.doi.org/10.1038/sj.gt.3302511

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1045259912

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/15815682


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