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在幼儿基因疗法的机器人辅助内输注:手术考虑

Rrita Daci1, Mohammed Salman Shazeeb2, Brittany Owusu-Adjei1

  • 11Department of Neurosurgery, UMass Chan Medical School.

Journal of neurosurgery. Pediatrics
|August 15, 2025
PubMed
概括

这项研究表明,使用腺关联病毒 (AAV) 载体的双边thalamic基因疗法在患有GM2性腺症的儿童中是安全和准确的. 通过对流增强输送 (CED) 技术,即使在婴儿身上,也可以准确地定位并在乳腺体内进行注.

关键词:
转基因二氧化碳 (GM2) 质化症通过对流增强的交付.基因治疗的基因疗法儿科的立体术神经外科手术.手术技术是指手术技术的使用.泰拉姆体内注射药物输液.

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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 inserted. The...