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Treatment of Macular Degeneration
TED-R

Embryonic Stem Cell-derived
Retinal Pigment Epithelium and Photoreceptor Cells

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Macular Degeneration
A disease in which vision is impaired or lost due to failure of the macula located in the center of the retina
Cause
Deterioration of functions of macular due to accumulation of hydrophobic waste resulted from aging, and genetic factors, toxicity, inflammation, etc.
Symptom

Early: Transient loss of vision

Mid-term: The lines of an object look bent like waves. With further development, the center of the object appears dark and is not visible, creating a blank space or the center of the object is not visible as if it had been erased

Terminal: Loss of vision

Basic Research Non-clinical Phase 1 Phase 2 Phase 3 Approval
Nonclinical trial
in progress
OUTLINE
Therapy
imageInject into degenerated macular imagemacular degeneration improvement
Technology
TED technology (using DM/SB)
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TED
 
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Isolation culture
DATA
Retinal Pigment Epithelium (RPE) Differentiation
Immunofluorescence staining of RPE cells

Differentiation into RPE cells was confirmed as proteins known to be specifically expressed in RPE cells, namely, ZO1, Mitf, Pax6, Bestrophin, and RPE65, were observed.

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    Mitf/ZO1

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    Pax6/ZO1

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    Bestrophin/DAPI

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    RPE65/DAPI

Confirmation of RPE cell function (1)

FITC-labeled latex beads and FITC-labeled photoreceptor outer segment were treated on differentiated RPE cells to observe phagocytosis, one of the main functions of RPE cells. As a result, an influx of fluorescent substances into RPE cells was observed. Thus, phagocytosis of differentiated RPE cells was confirmed.

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    ZO1/FITC-labeled latex beads

  • apical

    nuclei

    basal

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    ZO1/FITC-labeled photoreceptor outer segments

Confirmation of RPE cell function (2)

Through morphological verification of differentiated RPE cells using electron microscope, phagosome (A), a typical structure of RPE cells for phagocytosis, tight junction (A*) for cell-cell interactions, melanin granule (A**) for pigmentation and microvilli (B) used in phagocytosis were identified.

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Confirmation of RPE cell function (3)

It was confirmed that differentiated RPE cells secrete PEDF and VEGF, the growth factors typically secreted from mature RPE cells, at a level similar to that of the positive control group (adult RPE cell line).

  • 배경 배경
    그래프 그래프
  • 배경 배경
    그래프 그래프
Photoreceptor Differentiation
Immunofluorescence staining of photoreceptors

Differentiation into photoreceptors was confirmed as proteins known to be specifically expressed in photoreceptors, namely, blue opsin, red opsin, PDE6b, recoverin, and rhodopsin were observed.

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Confirmation of photoreceptor function

When photoreceptors undergo an optical transmission signal, the concentration of cyclic GMP (Guanosine Monophosphate) becomes lower in photopic vision than in scotopic vision. The experiment confirmed the function of differentiated photoreceptors as the cyclic GMP decreased more in photopic vision (light) than in scotopic vision (dark).

배경 배경
그래프 그래프
Macular Degeneration Improvement
  • Retinal thickness improvement

    When two types of TED-R cells were transplanted respectively into model animals with macular degeneration, the thickness of the outer nuclear layer (ONL), which became thinner due to macular degeneration, showed improvement compared to that of the control group.

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  • Visual performance improvement

    When two types of TED-R cells were transplanted respectively into model animals with macular degeneration, the visual performance showed improvement as it was observed that the electroretinogram (b-wave), which decreased due to macular degeneration, was higher than that of the control group.

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