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Updated: Jan 15, 2026

A Step-By-Step Method to Detect Neutralizing Antibodies Against AAV using a Colorimetric Cell-Based Assay
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基因疗法的长期安全性和有效性 对于腺氨酸脱氨酶缺乏症的基因疗法

Claire Booth1,2, Katelyn Masiuk3, Konstantinos Vazouras1,2

  • 1University College London Great Ormond Street Institute of Child Health, London.

The New England journal of medicine
|October 15, 2025
PubMed
概括

基因疗法为腺脱氨酶缺乏症 (ADA-SCID) 提供治愈治疗,这是一种严重的免疫疾病. 长期结果显示,在接受lentiviral基因治疗的患者中,疗效和安全性持续.

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相关概念视频

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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