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The role of complement membrane attack complex in dry and wet AMD - From hypothesis to clinical trials

Rajendra Kumar-Singh1

  • 1Department of Developmental, Molecular and Chemical Biology, Tufts University School of Medicine, Boston, MA, 02111, USA.

Experimental Eye Research
|May 15, 2019
PubMed

Insights

Constant complement system activation, forming the membrane attack complex (MAC), drives age-related macular degeneration (AMD). Inhibiting MAC with CD59 via gene therapy shows promise for treating both dry and wet AMD.

Area of Science:

  • Ophthalmology
  • Immunology
  • Genetics

Background:

  • Age-related macular degeneration (AMD) is linked to complement system overactivation.
  • The alternative complement pathway's constant 'tickover' deposits the membrane attack complex (MAC) on ocular tissues.
  • MAC, at sub-lytic or lytic levels, contributes to cellular dysfunction and death in AMD.

Purpose of the Study:

  • To review the role of MAC in dry and wet AMD pathophysiology.
  • To discuss the therapeutic potential of CD59, a MAC inhibitor, for AMD.
  • To outline the rationale for using adeno-associated virus (AAV)-delivered CD59 in AMD treatment.

Main Methods:

  • Review of existing data on complement pathway activation in AMD.
  • Analysis of CD59's inhibitory function on MAC assembly.
  • Examination of ongoing clinical trials using AAV-CD59 gene therapy for AMD.

Main Results:

  • Evidence supports MAC deposition as a key factor in AMD progression.
  • CD59 is a natural inhibitor of MAC, suggesting therapeutic value.
  • Gene therapy trials delivering CD59 are underway for dry and wet AMD.

Conclusions:

  • The complement system, specifically MAC, plays a critical role in both dry and wet AMD.
  • AAV-mediated CD59 gene therapy presents a promising therapeutic strategy for AMD.
  • Targeting MAC inhibition offers a novel approach to treating this vision-impairing disease.
Keywords:
Adeno-associated virusCD59ComplementGeographic atrophyMembrane attack complexage-related macular degenerationgene therapy

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