JCI - Small populations, big impact: leveraging rare disease gene therapies to benefit millions

Small populations, big impact: leveraging rare disease gene therapies to benefit millions

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Advancing genetic medicine for treatment of rare diseases.

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(A) A brief overview of gene therapy technologies and their potential applications in rare diseases. (B) Rare diseases are often on the front lines of innovation in precision medicine and gene therapy, establishing a foundation for broader use of these technologies.

Small populations, big impact: leveraging rare disease gene therapies to benefit millions

Small populations, big impact: leveraging rare disease gene therapies to benefit millions

Abstract

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