Article

Synthetic dosage-compensating miRNA circuits for quantitative gene therapy

2024-03-14

Abstract excerpt

A longstanding challenge in gene therapy is expressing a dosage-sensitive gene within a tight therapeutic window. For example, loss of MECP2 function causes Rett syndrome, while its duplication causes MECP2 duplication syndrome. Viral gene delivery methods generate variable numbers of gene copies in individual cells, creating a need for gene dosage-invariant expression systems. Here, we introduce a compact miRNA-b...

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Literature Corpus work: 477200d4-ca5e-530e-b83e-ecaff9a26bd3
DOI: 10.1101/2024.03.13.584179

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