13 lecture2 rd-amd therapies
TRANSCRIPT
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Emerging Therapies: Retinal & Macular Degenerations
PHAR 40040 (2013)
Dr. Breandn Kennedy
UCD Conway Institute
Refs:
"The Eye, Basic Sciences in Practice"
2nd Ed (Forrester, Dick, McMenamin, Lee, Saunders)
http://webvision.med.utah.edu/photo1.html
L1: Overview of Course Goals
L2: Vision
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Learning objectives:
1. Understand the hallmarks of retinal and macular degenerations
2. Understand the general therapeutic approaches to RD/MD (cell-,
drug- & gene-based)
3. Understand the logic behind use of these therapeutic approaches
for RD/MD and specific advantages/disadvantages of the eye
4. Display ability to critically evaluate the scientific/clinical evidenceon the effectiveness/potential of these approaches.
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I. The Human Eye
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Eyeenables visioni.e.capture of image of external surroundings
Environmental light
Detected by photoreceptors in retina
Signal to brain via optic nerve
Vision
Human Eye
-sphere ~2.5 cm in diameter-vol. of ~6.5 mls
-detects light of 400-700 nm
wavelengths
-the window to the brain
-an approachable part of thebrain
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Structural Arrangement of RETINAL Cells
http://webvision med utah edu/sretina html
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Two or three types of
cone photoreceptor and
a single type of rod
photoreceptor are
present in the normal
mammalian retina.
Some non-mammalian
retinas have even more
cone types
Rod & Cone
Photoreceptors
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Structure of a rod photoreceptor
phototransduction
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Cones are clustered in the
specialised foveal region of
the retina
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Molecular basis of how we see; photopigments
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-photopigment comprises 11-cis-retinal plus an opsin protein
-retina detects different wavelengths of light as different 11-cis-retinal:opsin
combos have different Abs. Max.
e.g. rod pigments = 11-cis-retinal + rhodopsin
blue pigments = 11-cis-retinal + blue cone opsin
Colour Vision
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ANALYSIS OF BLINDNESS
Functional ERG
Pupil Dilation
Behaviour/Obstacle Course Morphological
Histology
FundoscopyOCT
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Vision -------Retinal/Macular Degeneration -----Blindness
Goal: prevent or reverse blindness due to retinal degeneration
Emerging Approaches:
-1) Pharmacological
-2) Gene-Based
-3) Cell-Based
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ADDITIONAL RESOURCES
http://www.youtube.com/watch?v=VrEXkiTcyiU
http://www.youtube.com/watch?v=Fm45A4yjmvo
http://webvision.med.utah.edu/book/electrophysiology/the-
electroretinogram-clinical-applications/
http://www.youtube.com/watch?v=Fm45A4yjmvohttp://www.youtube.com/watch?v=Fm45A4yjmvohttp://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://webvision.med.utah.edu/book/electrophysiology/the-electroretinogram-clinical-applications/http://www.youtube.com/watch?v=Fm45A4yjmvohttp://www.youtube.com/watch?v=Fm45A4yjmvohttp://www.youtube.com/watch?v=Fm45A4yjmvo