Polmar S H
Ciba Found Symp. 1978(68):213-30. doi: 10.1002/9780470720516.ch14.
Addition of adenosine deaminase (ADA) restored in vitro responses of lymphocytes from a patient with ADA deficiency and severe combined immunodeficiency (SCID). Enzyme replacement therapy, using red blood cells as a source of encapsulated human ADA, restored both T and B cell function in this patient. Ten other ADA--SCID patients have been treated with this form of enzyme replacement and five have responded to therapy. Lymphocytes from ADA--SCID patients treated with enzyme replacement become immunocompetent but remain enzyme deficient. Studies of these cells provide evidence supporting both cyclic AMP- and dATP-mediated immunosuppressive mechanisms in ADA--SCID. These observations suggest that inhibition of cyclic AMP synthesis and/or deoxycytidine (and possibly thymidine) supplementation may be useful new biochemical approaches to the therapy of ADA--SCID.
添加腺苷脱氨酶（ADA）可恢复来自一名患有ADA缺乏症和严重联合免疫缺陷（SCID）患者的淋巴细胞的体外反应。使用红细胞作为包封人ADA来源的酶替代疗法恢复了该患者的T细胞和B细胞功能。其他10名ADA - SCID患者已接受这种形式的酶替代治疗，其中5名患者对治疗有反应。接受酶替代治疗的ADA - SCID患者的淋巴细胞具有免疫活性，但仍缺乏酶。对这些细胞的研究提供了支持ADA - SCID中由环磷酸腺苷（cAMP）和三磷酸脱氧腺苷（dATP）介导的免疫抑制机制的证据。这些观察结果表明，抑制环磷酸腺苷合成和/或补充脱氧胞苷（可能还有胸腺嘧啶核苷）可能是治疗ADA - SCID的有用的新生化方法。
