Hauswirth William W

Wassmer Sarah J, De Repentigny Yves, Sheppard Derek, Lagali Pamela S, Fang Lijun, Coupland Stuart G et al.

Gene therapy with XIAP protected retinal ganglion cells and optic nerves in a mouse model of LHON, preserving axon health and delaying disease progression.

Guy John, Feuer William J, Davis Janet L, Porciatti Vittorio, Gonzalez Phillip J, Koilkonda Rajeshwari D et al.

This study found low/medium-dose gene therapy for LHON was safe and improved vision in treated eyes, suggesting promise for further testing of higher doses.

Talla Venu, Koilkonda Rajeshwari, Porciatti Vittorio, Chiodo Vince, Boye Sanford L, Hauswirth William W et al.

NDUFA6 gene therapy in an MS model rescued mitochondrial function, preventing significant retinal ganglion cell loss and preserving vision. This suggests a potential treatment for optic neuritis.

Guy John, Qi Xiaoping, Koilkonda Rajeshwari D, Arguello Tania, Chou Tsung-Han, Ruggeri Marco et al.

AAV delivery of human ND4 to mouse eyes was safe, with proper mitochondrial integration and no RGC loss, suggesting potential for Leber hereditary optic neuropathy treatment.

Pease Mary Ellen, Zack Donald J, Berlinicke Cynthia, Bloom Kristen, Cone Frances, Wang Yuxia et al.

This study found CNTF gene therapy significantly protected retinal ganglion cells in experimental glaucoma, suggesting it's a promising neuroprotective strategy for human glaucoma.

Qi Xiaoping, Sun Liang, Lewin Alfred S, Hauswirth William W, Guy John

Antioxidant gene therapy in mice with optic neuritis significantly reduced retinal ganglion cell and axonal loss. This offers a promising long-term neuroprotective strategy for patients with optic neuritis and multiple sclerosis.

Qi Xiaoping, Lewin Alfred S, Sun Liang, Hauswirth William W, Guy John

Studying experimental optic neuritis, researchers found that suppressing mitochondrial oxidative stress via SOD2 gene delivery significantly reduced optic nerve damage and ganglion cell loss long-term, suggesting a neuroprotective therapeutic strategy.

Qi Xiaoping, Sun Liang, Lewin Alfred S, Hauswirth William W, Guy John

Mutant human ND4 gene expression in mice replicated LHON's optic neuropathy, causing retinal ganglion cell death. Normal ND4 was safe, suggesting potential LHON gene therapy.

Martin Keith R G, Quigley Harry A, Zack Donald J, Levkovitch-Verbin Hana, Kielczewski Jennifer, Valenta Danielle et al.

Gene therapy delivering BDNF via AAV protected retinal ganglion cells in a rat glaucoma model, suggesting neurotrophic therapy could complement IOP lowering in human glaucoma treatment.

Qi Xiaoping, Lewin Alfred S, Hauswirth William W, Guy John

Reducing mitochondrial antioxidant SOD2 in mice induced optic neuropathy mimicking Leber hereditary optic neuropathy (LHON). This suggests reactive oxygen species are key in LHON's pathogenesis, offering a model for studying oxidative optic nerve injury.