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Molecular Mechanisms of Neurodegeneration and Neuroprotection for Retinal and Optic Nerve Diseases

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Neurobiology".

Deadline for manuscript submissions: 20 August 2025 | Viewed by 790

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Department of Ophthalmology and Visual Science, Chiba University Graduate School of Medicine, Inohana 1-8-1, Chuo-ku, Chiba 260-8670, Japan
Interests: neuroprotection; neuroprotective therapy; diabetic retinopathy; optic nerve
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Special Issue Information

Dear Colleagues,

Neuronal cell death is an irreversible change and associated with the pathogenesis of all retinal and optic nerve diseases. Because neuronal cell death is directly related to vision loss, neuronal cell death must be protected before the point of no return. A variety of types of cell death, such as necrosis, apoptosis, pyroptosis, autophagic cell death, ferroptosis, or necroptosis, can be associated with the pathogenesis of retinal and optic nerve diseases. Thus, multiple neuroprotective therapies against various types of cell death may be required for retinal and optic nerve diseases. To establish neuroprotective therapies, the precise mechanism of neuronal cell death must be elucidated. Otherwise, we cannot identify where the point of no return is.

This Special Issue focuses on the molecular mechanisms of neuronal cell death as well as neuroprotective and regenerative therapies for retinal and optic nerve diseases. This Special Issue covers all studies from bench to bed, and thus clinical studies and trials for neuroprotective therapies are also welcome. We hope that this Special Issue contributes to the development of medical science more than a little.

Prof. Dr. Toshiyuki Oshitari
Guest Editor

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Keywords

  • neuroprotection
  • neurodegeneration
  • neuronal cell death
  • regeneration
  • cell death and survival pathways
  • neurovascular unit
  • oxidative stress
  • inflammation
  • advanced glycation end products
  • ischemia
  • optic nerve injuries
  • glaucoma
  • optic neuritis
  • retinitis pigmentosa
  • diabetic retinopathy
  • age-related macular degeneration
  • retinal vein/artery occlusion
  • gene therapy
  • cell plantation
  • nutrients
  • neuroprotectants
  • drug delivery
  • translational research

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Published Papers (1 paper)

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17 pages, 3732 KiB  
Opinion
Repurposing Dimethyl Fumarate Targeting Nrf2 to Slow Down the Growth of Areas of Geographic Atrophy
by Serge Camelo
Int. J. Mol. Sci. 2025, 26(13), 6112; https://doi.org/10.3390/ijms26136112 - 25 Jun 2025
Viewed by 369
Abstract
Recently, marketing authorizations were granted by the Federal Drug Administration (FDA) for pegcetacoplan and avacincaptad pegol, which inhibit C3 and C5 complement components, respectively. These two drugs were demonstrated to slow down the growth of atrophic areas in the retina. These authorizations represent [...] Read more.
Recently, marketing authorizations were granted by the Federal Drug Administration (FDA) for pegcetacoplan and avacincaptad pegol, which inhibit C3 and C5 complement components, respectively. These two drugs were demonstrated to slow down the growth of atrophic areas in the retina. These authorizations represent a huge breakthrough for patients suffering from geographic atrophy (GA), the late stage of the dry form of Age-related Macular Degeneration (AMD). Until then, no treatment was available to treat this blinding disease. However, these two new compounds inhibiting the complement system are still not available for patients outside of the United States, and they are not devoid of drawbacks, including a poor effect on vision improvement, an increased risk of occurrence of the neovascular form of AMD and the burden of patients receiving recurrent intravitreal injections. Thus, the important medical need posed by GA remains incompletely answered, and new therapeutic options with alternative modes of action are still required. Oxidative stress and inflammation are two major potential targets to limit the progression of atrophic retinal lesions. Dimethyl fumarate, dimethyl itaconate and other activators of the transcription factor nuclear factor erythroid 2-related factor 2 (Nrf2) display antioxidants and immunomodulatory properties that have shown evidence of efficacy in in vitro and in vivo models of dry AMD. Tecfidera®, whose active principle is dimethyl fumarate, is already commercialized for the treatment of autoimmune diseases such as multiple sclerosis and psoriasis. The aim of this review is to present the rationale and the design of the clinical trial we initiated to test the effectiveness and safety of repurposing Tecfidera®, which could represent a new therapeutic alternative in patients with the dry form of AMD. Full article
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