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Animal models for ocular gene therapies
Author(s) -
Arsenijevic Y.,
Kostic C.
Publication year - 2016
Publication title -
acta ophthalmologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.534
H-Index - 87
eISSN - 1755-3768
pISSN - 1755-375X
DOI - 10.1111/j.1755-3768.2016.0161
Subject(s) - computational biology , disease , retinal degeneration , biology , phenotype , human disease , neuroscience , bioinformatics , gene , genetics , medicine , pathology
Summary Disease‐causing variants of a large number of genes trigger inherited retinal degeneration leading to photoreceptor loss. Because cones are essential for daylight and central vision such as reading, mobility and face recognition, the presentation will focus on a variety of animal models for cone diseases. The pertinence of using these models to reveal genotype/phenotype correlations and to evaluate new therapeutic strategies will bediscussed. Interestingly, several large animal models recapitulate human diseases and can serve as a strong base to study the disease biology and to assess the scale‐up of new therapies. Examples of innovative approaches will be presented such as lentiviral‐based transgenesis in pigs. The models we will discuss permit to explore common mechanisms existing between different species and to highlight the pathways that may be specific to primates, including human.