Genes & CellsGenes & CellsГены и КлеткиGenes and Cells2313-18292500-2562Human Stem Cells Institute12156410.23868/gc121564ArticlesСтатьиGene and cell therapy of retinal diseasesГенная и клеточная терапия заболеванийсетчатки глазаMaximovV VМаксимовВ В

Vavilov Institute of General Genetics of RAS, Moscow

Институт общей генетики им. Н.И. Вавилова РАН, Москва

LagarkovaM AЛагарьковаМ А

Vavilov Institute of General Genetics of RAS, Moscow

Институт общей генетики им. Н.И. Вавилова РАН, Москва

KiselevS LКиселевС Л

National Research Centre «Kurchatov Institute», Moscow

Национальный исследовательский центр «Курчатовский институт», Москва

Vavilov Institute of General Genetics of RAS, MoscowИнститут общей генетики им. Н.И. Вавилова РАН, МоскваNational Research Centre «Kurchatov Institute», MoscowНациональный исследовательский центр «Курчатовский институт», Москва1509201273NO3 (2012)№3 (2012)122011012023Copyright ©; 2012, Eco-VectorCopyright ©; 2012, Эко-Вектор2012Eco-VectorЭко-Векторhttps://genescells.ru/2313-1829/article/view/121564

Gene and cell therapy are tightly associated and quickly developing areas of biomedicine, which purpose is development of methods to cure diseases caused by genetic defects and/or death of certain cell types. Current state of methods of retinal diseases gene and cell therapy is analyzed in this review. We reviewed development of gene therapeutic approaches to treatment of the 2-nd form of Lebers congenital amaurosis and other retinal diseases. We also compared therapeutic potential of pluripotent stem cells and human adult retinal stem cells. Therapeutic potential of pluripotent stem cells seems to be better due to their ability for unlimited expansion and organogenesis.

Генная и клеточная терапия заболеваний - это тесно взаимосвязанные и быстро развивающиеся области биомедицины, целью которых является разработка методов лечения заболеваний, в основе которых лежат генетические дефекты и(или) гибель определённых клеточных типов. В этом обзоре проанализировано современное состояние методов генной и клеточной терапии заболеваний сетчатки глаза. Особое внимание уделено развитию генно-терапевтических подходов к ечению 2-й формы врождённого амавроза Лебера и других заболеваний сетчатки, а также сравнению терапевтического потенциала плюрипотентных стволовых клеток и стволовых клеток сетчатки глаза взрослого человека. Терапевтический потенциал плюрипотентных клеток представляется предпочтительней вследствие их способности к неограниченной экспансии и органогенезу.

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