Preclinical Assessment of Mitochondrial-Targeted ND4 Gene Therapy for Leber Hereditary Optic Neuropathy.

چکیده

The purpose of this study was to establish preclinical safety data to advance mitochondrial targeted AAV-delivered human ND4 (MTSAAV/hND4) for treatment of Leber Hereditary Optic Neuropathy (LHON). MTSAAV/hND4 was bilaterally injected into mouse eyes to evaluate potential adverse effects on visual…

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موضوعات مرتبط

• تصویربرداری و تشخیص