Biopolym. Cell. 2013; 29(1):21-32.
http://dx.doi.org/10.7124/bc.000802
Огляди
Генна терапія, заснована на стовбурових клітинах
1Назір З., 1Іршад С.
  1. Інститут біохімії і біотехнології, Университета Пенждаба
    Лахор, Пакистан

Abstract

Стовбурові клітини мають величезний потенціал для регенеративної медицини. Протягом десятиліть було доведено безпеку і ефективність терапії на основі HSC, у той час як використання MSC, обмежене строками зберігання клітин та деякими іншими причинами, лише в окремих експериментах давало позитивні наслідки з короткочасною дією, які потребують подальшого вивчення. Незважаючи на те, що ІПСК залишаються основним джерелом при терапії стовбуровими клітинами, як і раніше, існують серйозні питання біобезпеки, які потрібно враховувати особливо в разі клітин, що продаються для клінічних випробувань. Тим не менш, найперспективнішими для регенеративної медицини майбутнього є лінії стовбурових клітин, створені для конкретного пацієнта, а також коригування гомологічних генів і вбудовування трансгена в певне місце в геномі для подальшої експресії і диференціювання. Крім того, розвиток рекомбінантної аденовірусної технології (rAAV) і використання нуклеази цинкового пальця (ZFN) дозволяє вважати гомологічну рекомбінацію найкращим інструментом для генної терапії, заснованої на стовбурових клітинах.
Keywords: стовбурові клітини, МСК, ІПСК, ZFN, rAAV, корекція гомологічних генів, регенеративна медицина
Повний текст: (PDF, англійською)

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