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AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway

25 Oct 2021

Abstract excerpt

Genome editing in the lung has the potential to provide long-term expression of therapeutic protein to treat lung genetic diseases. Yet efficient delivery of CRISPR to the lung remains a challenge. The NIH Somatic Cell Genome Editing (SCGE) Consortium is developing safe and effective methods for genome editing in disease tissues. Methods developed by consortium members are independently validated by the SCGE...

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PMID: 34695545
DOI: 10.1016/j.ymthe.2021.10.023

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