Neuroglobin gene therapy prevents optic atrophy and preserves durably visual function in Harlequin mice.

Neuroglobin (NGB) is considered as an endogenous neuroprotective molecule against stroke, since the protein alleviates the adverse effects of hypoxic and ischemic insults. We previously demonstrated the functional link between NGB and mitochondria since it is required for respiratory chain function. Thus, here, we evaluated the relevance of this effect in the Harlequin (Hq) mouse strain, which exhibits retinal ganglion cell (RGC) loss and optic atrophy due to a respiratory chain complex I (CI) defect. A twofold decrease of NGB amounts was observed in Hq retinas. We constructed a recombinant adeno-associated virus which combines to the mouse NGB open reading frame, its 5' and 3'UTR, for guarantying mRNA stability and translation capacity. The vector was administrated intravitreally to Hq mice and NGB expression was stable for up to 7 months without negative effect on retinal architecture or function. On the contrary, RGCs and their axons were substantially preserved from degeneration; consequently, CI activity in optic nerves was protected conferring improvements in vision. Hence, we established that NGB prevents respiratory chain impairment, therefore, protecting visual function otherwise compromised by mitochondrial energetic failure.

[Results of the Collaborative Initial Glaucoma Treatment Study (CIGTS)]. / Ergebnisse der "Collaborative Initial Glaucoma Treatment Study" (CIGTS).

The Collaborative Initial Glaucoma Treatment Study (CIGTS) was initiated to evaluate whether local medical therapy or trabeculectomy is the better initial treatment for patients with newly diagnosed open-angle glaucoma. Visual field progression, intraocular pressure, and visual acuity were to be monitored for a period of 5 years. A total of 607 patients were randomly distributed among the two groups and controlled at 6-month intervals. At the same time the investigators tried to record quality of life using a questionnaire. Both trabeculectomy and medical therapy were able to lower intraocular pressure significantly, whereas trabeculectomy was more effective. The results of the questionnaire showed only minor differences between the two groups. The medically treated patients reported slightly more ocular symptoms than the patients in the trabeculectomy group.

Reversal of optic canal stenosis in osteopetrosis after bone marrow transplant.

PURPOSE: To describe a patient with infantile osteopetrosis and optic atrophy secondary to optic canal stenosis who demonstrated optic canal enlargement after bone marrow transplant. METHODS: Case report. A 3-month-old infant with infantile "malignant" osteopetrosis underwent ophthalmic examination, including visual evoked potentials, electroretinogram, and computed tomography (CT). Bone marrow transplant was performed at 8 months of age. RESULTS: Examination revealed visual loss and optic atrophy, left eye greater than right eye, secondary to optic canal stenosis. Flash visual evoked potentials revealed a normal waveform in both eyes with increased latency in the left eye. Electroretinogram was normal in both eyes. CT after bone marrow transplant showed enlargement of the optic canals. Vision remains stable 43 months after bone marrow transplant. CONCLUSIONS: Bone marrow transplant in infantile osteopetrosis may be followed by reversal of optic canal stenosis and preservation of vision.