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Published on: August 7, 2015
The Complement System as a Therapeutic Target in Retinal Disease
Joshua Ong1, Arman Zarnegar2, Amrish Selvam2
1Department of Ophthalmology and Visual Sciences, University of Michigan Kellogg Eye Center, Ann Arbor, MI 48105, USA.
Insights
The complement system is crucial for retinal health but its dysregulation causes diseases like geographic atrophy. Therapies targeting this system are now available, offering new hope for treating retinal conditions.
Area of Science:
- Ophthalmology
- Immunology
- Molecular Biology
Background:
- The complement cascade is essential for host defense and maintaining retinal integrity.
- Dysregulation of the complement system is implicated in retinal pathologies such as geographic atrophy and diabetic retinopathy.
- The aging process can affect complement function, increasing susceptibility to retinal diseases.
Purpose of the Study:
- To discuss the role of complement system dysfunction in retinal pathophysiology.
- To review cellular, animal, and clinical studies targeting the complement system for retinal diseases.
- To provide an overview of approved complement inhibitors for geographic atrophy.
Main Methods:
- Review of existing literature on complement system in retinal diseases.
- Analysis of data from in vivo, ex vivo, and clinical trials.
- Examination of approved therapeutic agents targeting the complement cascade.
Main Results:
- Complement system dysregulation is a key factor in the development of specific retinal pathologies.
- Therapeutic strategies targeting complement components have shown promise in preclinical and clinical studies.
- Several complement inhibitors are now FDA-approved for treating geographic atrophy.
Conclusions:
- The complement system represents a significant therapeutic target for various retinal diseases.
- Further research is needed to fully elucidate complement mechanisms and optimize treatment strategies.
- Targeting the complement cascade offers a promising avenue for managing and treating debilitating retinal conditions.
Abstract:
The complement cascade is a vital system in the human body's defense against pathogens. During the natural aging process, it has been observed that this system is imperative for ensuring the integrity and homeostasis of the retina. While this system is critical for proper host defense and retinal integrity, it has also been found that dysregulation of this system may lead to certain retinal pathologies, including geographic atrophy and diabetic retinopathy. Targeting components of the complement system for retinal diseases has been an area of interest, and in vivo, ex vivo, and clinical trials have been conducted in this area. Following clinical trials, medications targeting the complement system for retinal disease have also become available. In this manuscript, we discuss the pathophysiology of complement dysfunction in the retina and specific pathologies. We then describe the results of cellular, animal, and clinical studies targeting the complement system for retinal diseases. We then provide an overview of complement inhibitors that have been approved by the Food and Drug Administration (FDA) for geographic atrophy. The complement system in retinal diseases continues to serve as an emerging therapeutic target, and further research in this field will provide additional insights into the mechanisms and considerations for treatment of retinal pathologies.

