Principles of and strategies for germline gene therapy

Don P. Wolf, Paul A. Mitalipov, Shoukhrat M. Mitalipov

    Research output: Contribution to journalReview article

    7 Scopus citations

    Abstract

    Monogenic disorders occur at a high frequency in human populations and are commonly inherited through the germline. Unfortunately, once the mutation has been transmitted to a child, only limited treatment options are available in most cases. However, means of correcting disease-causing nuclear and mitochondrial DNA mutations in gametes or preimplantation embryos have now been developed and are commonly referred to as germline gene therapy (GGT). We will discuss these novel strategies and provide a path forward for safe, high-efficiency GGT that may provide a promising new paradigm for preventing the passage of deleterious genes from parent to child.

    Original languageEnglish (US)
    JournalNature medicine
    DOIs
    StatePublished - Jan 1 2019

    ASJC Scopus subject areas

    • Biochemistry, Genetics and Molecular Biology(all)

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