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GENE THERAPY FOR OCULAR IMPROVEMENT

机译:进行基因改良的基因疗法

摘要

Targeted non-surgical administration of a nucleic acid formulation to the suprachoroidal space (SCS) of the eye of a human subject permits effective treatment of ocular disorders, including posterior ocular or choroidal maladies. In one embodiment, the method comprises inserting a hollow microneedle into the eye at an insertion site and infusing a nucleic acid formulation through the inserted microneedle and into the suprachoroidal space of the eye. The infused nucleic acid formulation flows within the suprachoroidal space away from the insertion site. In one embodiment, the fluid nucleic acid formulation comprises nucleic acid nanoparticles consisting of one molecule of nucleic acid.
机译:对人类受试者的眼的脉络膜上腔(SCS)的靶向非手术给药可以有效治疗眼部疾病,包括后眼或脉络膜疾病。在一个实施方案中,该方法包括将中空微针在插入部位插入眼睛中,并通过所插入的微针将核酸制剂注入眼睛的脉络膜上腔中。注入的核酸制剂在脉络膜上腔内流离插入部位。在一实施方案中,流体核酸制剂包含由一分子核酸组成的核酸纳米颗粒。

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