CAR-T 세포면역요법의 기본원리는 유전공학적(genetic engineering) 수단을 이용하여 T세포 표면에 CAR분자를 표현하는 것입니다. CAR-T세포는 그 표면에 발현된 scFv를 통해 특이하게 종양세포 표면의 항원을 인식해 종양세포를 살상할 수 있습니다.
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Mucopolysaccharidosis IVA (MPS IVA) - also known as Morquio Syndrome, type A - is inherited in an autosomal recessive manner and caused by mutations in the GALNS gene which lead to N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency, resulting in the accumulation of keratan sulfate (KS) and chondroitin-6-sulfate (C6S).
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