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This chapter discusses trials in which gene therapy is used to treat Gaucher disease. Studies include trials of gene therapy use in patients with both Type I Gaucher disease and Type II Gaucher disease. Studies include recruiting, active, and approved for marketing trials.

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Gaucher 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 Gaucher disease. Studies include trials of gene therapy use in patients with both Type I Gaucher disease and Type II Gaucher disease. Studies include recruiting, active, and approved for marketing trials.