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lenadogene nolparvovec (Lumevoq)
Indications:
- gene therapy for Leber hereditary optic neuropathy (LHON) with MT-ND4 gene mutation*
* not FDA-approved
Dosage:
- intravitreal injection
Mechanism of action:
- uses adeno-associated virus vector to deliver wild-type MT-ND4 gene directly to the mitochondrial membrane of retinal ganglion cells.
General
gene therapy
ophthalmic agent (eyedrops)
References
- Davenport L
Gene Therapy Promising for Reversal of Hereditary Vision Loss.
Medscape. June 13, 2023
https://www.medscape.com/viewarticle/994339