TY - JOUR
T1 - Treatment of retinal and choroidal degenerations and dystrophies
T2 - Current status and prospects for gene-based therapy
AU - Weleber, Richard G.
AU - Kurz, Daryl E.
AU - Trzupek, Karmen M.
N1 - Funding Information:
This work was supported by a grant from the Foundation Fighting Blindness, Inc., and Research to Prevent Blindness.
PY - 2003/12
Y1 - 2003/12
N2 - Inherited retinal and choroidal degenerations account for a significant portion of blindness in children and young adults. This article reviews the current status and future prospects for the treatment of these disorders. Current treatment strategies include nutritional intervention for gyrate atrophy of the choroid and retina with hyperornithinemia, abetalipoproteinemia, and Refsum's disease, as well as vitamin A supplementation for retinitis pigmentosa. Future therapeutic prospects include gene therapy for both recessive and dominant disease, secondary gene-based therapies, such as pharmaceutic gene product replacement and treatment with survival factors, anti-apoptotic agents, and calcium blockers, and, finally, stem cell therapy.
AB - Inherited retinal and choroidal degenerations account for a significant portion of blindness in children and young adults. This article reviews the current status and future prospects for the treatment of these disorders. Current treatment strategies include nutritional intervention for gyrate atrophy of the choroid and retina with hyperornithinemia, abetalipoproteinemia, and Refsum's disease, as well as vitamin A supplementation for retinitis pigmentosa. Future therapeutic prospects include gene therapy for both recessive and dominant disease, secondary gene-based therapies, such as pharmaceutic gene product replacement and treatment with survival factors, anti-apoptotic agents, and calcium blockers, and, finally, stem cell therapy.
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U2 - 10.1016/S0896-1549(03)00072-5
DO - 10.1016/S0896-1549(03)00072-5
M3 - Review article
C2 - 14740999
AN - SCOPUS:0347595514
SN - 0896-1549
VL - 16
SP - 583
EP - 593
JO - Ophthalmology Clinics of North America
JF - Ophthalmology Clinics of North America
IS - 4
ER -