The lack of the N-alpha-glucosaminidase (Naglu) is responsible for the incidence of a rare disease - mucopolysaccharidosis, type IIIB (MPS IIIB). To date, studies have been conducted based on cells derived from patients suffering from MPS or using in vivo MPS mouse models. These limitations have allowed for defining our research goal - to create and characterize the first in vitro murine cellular MPS IIIB model. In the current work we present a new, stable cell line with confirmed accumulation of glycosaminoglycans. The line stability was achieved by immortalization using a lentivirus carrying the T-antigens of SV40. The Naglu-/- cells were confirmed to produce no Naglu enzyme. To confirm the proper functioning of the in vitro MPS IIIB model, we determined the activity and expression of cystathionine γ-lyase, rhodanese and 3-mercaptopyruvate sulfurtransferase, as well as the level of low molecular-weight thiols (reduced and oxidized glutathione, cysteine and cystine). The results were referred to our earlier findings originating from the studies on the tissues of the Naglu-/- mice that were used to create the lines. The results obtained in the Naglu-/- cells were in accordance with the results found in the mouse model of MPS IIIB. It suggests that the presented murine Naglu-/- cell lines might be a convenient in vitro model of MPS IIIB.

中文翻译：

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粘多糖贮积症的鼠类细胞模型，IIIB型（MPS IIIB）-一项初步研究，尤其着重于非氧化性L-半胱氨酸代谢。

缺乏N-α-氨基葡萄糖苷酶（Naglu）导致罕见疾病-粘多糖贮积症，IIIB型（MPS IIIB）的发生。迄今为止，已经基于源自患有MPS的患者的细胞或使用体内MPS小鼠模型进行了研究。这些限制使我们可以确定研究目标-创建并表征第一个体外鼠类细胞MPS IIIB模型。在当前的工作中，我们提出了一种新的，稳定的细胞系，其中已证实糖胺聚糖的积累。通过使用携带SV40 T-抗原的慢病毒永生化来获得品系稳定性。证实Naglu-/-细胞不产生Naglu酶。为了确认体外MPS IIIB模型的正常功能，我们确定了胱硫醚γ-裂解酶的活性和表达，罗丹酸和3-巯基丙酮酸硫转移酶，以及低分子量硫醇的水平（还原和氧化的谷胱甘肽，半胱氨酸和胱氨酸）。该结果参考了我们先前的发现，这些发现源自对用于创建系的Naglu-/-小鼠组织的研究。在Naglu-/-细胞中获得的结果与在MPS IIIB小鼠模型中发现的结果一致。这表明所提出的鼠Naglu-/-细胞系可能是MPS IIIB的方便的体外模型。在Naglu-/-细胞中获得的结果与在MPS IIIB小鼠模型中发现的结果一致。这提示所提出的鼠Naglu-/-细胞系可能是MPS IIIB的方便的体外模型。在Naglu-/-细胞中获得的结果与在MPS IIIB小鼠模型中发现的结果一致。这表明所提出的鼠Naglu-/-细胞系可能是MPS IIIB的方便的体外模型。