University of Miami Miller School of Medicine Researchers Describe Recent Advances in Gene Therapy (Mitochondrially Targeted Gene Therapy Rescues Visual Loss in a Mouse Model of Leber's Hereditary Optic Neuropathy).

Předmět:	GENE therapy RESEARCH personnel VISUAL training LABORATORY mice GENE targeting
Zdroj:	Gene Therapy Weekly; 12/25/2023, p2733-2733, 1p
Abstrakt:	A recent study conducted by researchers at the University of Miami Miller School of Medicine has shown promising results in the field of gene therapy for Leber's hereditary optic neuropathy (LHON), a mitochondrial genetic disease that causes irreversible blindness. The study focused on delivering the wildtype human NADH ubiquinone oxidoreductase subunit 4 (hND4) gene using mito-targeted AAV (MTSAAV) to rescue LHON mice. The results demonstrated improved retinal visual acuity and increased functional hyperemia response in the mice that were rescued with the wildtype hND4 gene. These findings suggest that MTSAAV-delivered gene therapy has the potential to treat LHON and improve visual impairment. [Extracted from the article]
Databáze:	Complementary Index
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