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通过纳米平台向肺部传递基因编辑技术的最新进展

Moritz Marschhofer¹, Simone P Carneiro¹, Olivia M Merkel¹

¹Department of Pharmacy, Pharmaceutical Technology and Biopharmaceutics, Ludwig-Maximilians-University, Munich, Germany.

Nanomedicine (London, England)

|August 22, 2025

概括

No abstract available in PubMed .

关键词:

在CRISPR/Cas9中其他国家吸入方式纳米粒子肺部输送

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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...