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Updated: Jun 18, 2025

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research
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Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research

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光遗传学和针对视网膜疾病的基因治疗:揭示基本原理,应用和未来前景

Merve Kulbay1, Nicolas Tuli2, Arjin Akdag2

  • 1Department of Ophthalmology & Visual Sciences, McGill University, Montreal, QC H4A 3S5, Canada.

Journal of clinical medicine
|July 27, 2024
PubMed
概括

光遗传学和基因疗法通过恢复细胞功能为遗传性视网膜疾病提供了新的治疗方法. 本综述详细介绍了它们的原理,应用以及眼科医生的临床潜力.

关键词:
遗传性视网膜疾病 遗传性视网膜疾病视觉遗传学 视觉遗传学有针对性的基因治疗.

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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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