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This chapter discusses trials in which gene therapy is used to treat Krabbe disease. Studies include the use of gene therapy in infant and late infant manifestations of Krabbe disease as well as long-term studies on gene therapy use in Krabbe disease patients.

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Krabbe Disease

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  • 发表日期：

    2025年

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  • Jeffrey N. Weiss

摘要

This chapter discusses trials in which gene therapy is used to treat Krabbe disease. Studies include the use of gene therapy in infant and late infant manifestations of Krabbe disease as well as long-term studies on gene therapy use in Krabbe disease patients.