13 lecture2 rd-amd therapies

8/12/2019 13 Lecture2 RD-AMD Therapies

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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

13 lecture2 rd-amd therapies

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