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Drugs of the Future
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Drugs Fut 2018, 43(12): 901
ISSN 0377-8282
e-ISSN 2013-0368
Copyright 2018 Clarivate
CCC: 0377-8282
DOI: 10.1358/dof.2018.043.12.2903218
Therapeutic targets for retinitis pigmentosa
Sorbera, L.A., Dulsat, C., Graul, A.I.
Retinitis pigmentosa (RP) is a heterogeneous group of inherited retinal dystrophies which result in progressive degeneration of photoreceptor cells leading to vision loss and eventually blindness. RP is a rod-cone dystrophy characterized by the progressive degeneration and loss of photoreceptors, usually beginning in the mid-periphery of the fundus and extending toward the macula and fovea. The earliest symptom is typically night blindness, followed by progressive loss of peripheral vision (i.e., tunnel vision) and eventual loss of central vision. There are no drugs approved for the treatment of RP, and all pharmacotherapy is used off-label. Because iron-associated oxidative stress has been implicated in the pathogenesis of RP, particularly in the process of cone degradation, available pharmacotherapy includes antioxidants (i.e., vitamin A) and omega-3 fatty acids (i.e., docosahexaenoic acid). In addition, pharmacological chaperones are also used. These are a diverse group of drugs that are able to bind to and stabilize misfolded proteins. Other therapeutic options include inhibitors of photoreceptor apoptosis, Maxi-K+ channel openers, gene therapy (i.e., CRISPR/Cas9 genome editing) and photoreceptor transplant or replacement. Researchers actively continue to search for effective treatments for RP with special attention given to the identification of novel targets for therapeutic intervention. This article presents those drug targets that are currently under active investigation for the treatment of RP.

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