The application of AAV virus in ophthalmic gene therapy has attracted wide attention from all walks of life. 9 patients with Leber hereditary optic neuropathy received intravitreous injection of rAAV2-ND4 for 3 years. Visual acuity was significantly improved after injection, and no serious safety problems were found in patients with LHON. European and American counterparts also tested the breakthrough. With adeno-associated virus particles restarting, gene therapy is no longer out of reach.
Leber hereditary optic neuropathy (LHON) is a blinding disease. Previous reports have shown that gene therapy has been successful and is likely to bring about significant progress in the treatment of LHON.
A team reported a prospective, open label trial involving nine patients with LHON. The study took 3 years and 4 months to investigate the long-term results of LHON gene therapy. Nine patients voluntarily received intravitreous injection of rAAV2-ND4.
The safety and efficacy of the gene therapy were determined by systematic examination and visual function test during 36 months follow-up. On the basis of successful LHON animal model trial, one patient received rAAV2-ND4 injection to the other eye 12 months after monocular gene therapy. The main results of this study were visual acuity recovery, visual field changes, visual evoked potential (VEP), visual coherence tomography, liver and kidney function, and antibodies against AAV2. Eight patients received unilateral gene therapy and improved visual function was found in bilateral eyes (patient 4, 6, 7 and 8) and untreated eyes (2, 3, 4, 6, 6 and 8). Visual regression fluctuations were observed in patients 2 and 9. The age of onset, course of disease and residual optic nerve fibers had no significant effect on the improvement of visual function. Visual field and visual evoked potential (VEP) were also improved. Binocular gene therapy patients (patient 1) improved their visual acuity after the first treatment. However, three months after he received gene therapy for another eye, the vision of the first eye declined. Animal experiments showed that ND4 was expressed stably in contralateral eyes after intravitreal injection.
AAV service in this experiment again made a great contribution!
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