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Updated: Jun 26, 2025

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精准医学基础设施:使罕见疾病的基因疗法在实践中可行

Sarah Wadmann1, Anna Brueckner Johansen1, Alfred Peter Born2

  • 1The Danish Center for Social Science Research - VIVE, Herluf Trolles Gade 11, DK-1052 Copenhagen, Denmark.

Social science & medicine (1982)
|May 17, 2024
PubMed
概括

精准医学,包括基因疗法,需要大量的实践工作才能融入医疗保健系统. 这涉及调整法规,政策和日常临床实践,重塑整个医疗保健部门的角色.

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Gene Therapy00:59

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...
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