Новые возможности терапии болезни Штаргардта

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Болезнь Штаргардта — наследственная дистрофия сетчатки, ассоциированная с мутациями в гене ABCA4. В настоящее время этиопатогенетических препаратов и методов лечения болезни Штаргардта, полностью прошедших клинические испытания, не существует. В обзоре обобщены экспериментальные и клинические исследования препаратов, направленных на снижение накопления димеров витамина A, липофусцина, ингибирование комплемента и на регенерацию ретинального пигментного эпителия с помощью трансплантации стволовых клеток, а также исследования по генотерапии с интравитреальным векторным введением функционального гена ABCA4.

Ключевые слова:

болезнь Штаргардта

дистрофия сетчатки

ABCA4 ген

ретинальный пигментный эпителий

зрительный цикл

липофусцин

фармакологическая терапия

стволовые клетки

генотерапия

Авторы:

Жоржоладзе Н.В.

ФГБНУ «Научно-исследовательский институт глазных болезней» Минобрнауки России

Шеремет Н.Л.

ФГБНУ «Научно-исследовательский институт глазных болезней» Минобрнауки России

Танас А.С.

ФГБНУ «Медико-генетический научный центр имени академика Н.П. Бочкова»

Стрельников В.В.

ФГБНУ «Медико-генетический научный центр имени академика Н.П. Бочкова»

Даты поступления:

12.05.2020

Даты принятия в печать:

17.06.2020

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