麝香- caart:一种新的精确细胞治疗肌肉特异性酪氨酸激酶重症肌无力

A. Payne, Sangwook Oh
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引用次数: 0

摘要

嵌合自身抗体受体(CAAR) T细胞是一种新型的基因工程T细胞免疫疗法,旨在持久地消除抗原特异性B细胞,同时保留健康的B细胞,理想情况下通过一次性输注导致B细胞介导的自身免疫性疾病的安全和持久缓解。我们描述了肌肉特异性酪氨酸激酶CAART细胞(麝香- caart)用于治疗麝香重症肌无力的临床前开发,麝香重症肌无力是一种使人衰弱的自身抗体介导的疾病,可能导致危及生命的肌肉无力。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
MuSK-CAART: A novel precision cellular therapy for muscle-specific tyrosine kinase myasthenia gravis
Chimeric autoantibody receptor (CAAR) T cells are a novel genetically-engineered T cell immunotherapy that aims to durably eliminate antigen-specific B cells while sparing healthy B cells, ideally leading to safe and lasting remission of B cell-mediated autoimmune diseases with a one-time infusion. We describe the preclinical development of muscle-specific tyrosine kinase CAAR T cells (MuSK-CAART) for the treatment of MuSK myasthenia gravis, a debilitating autoantibody-mediated disease that causes potentially life-threatening muscle weakness.
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