Therapy for polyglutamine (POLYQ) diseases
A polyglutaminic acid, disease technology, applied in the field of treatment options for polyglutaminic acid disease, can solve the problem that there is no effective medical treatment or potential cure for SCA
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example 1
[0109] Example 1 QPSC changes the phenotype of SCA3
[0110] QPSCs alter the phenotype of SCA3 mice. Such as figure 1 As shown, SCA3 mice display a slightly wider base compared to wild-type mice and after QPSC treatment, the appearance of SCA3 mice looks similar to wild-type mice. In various functional tests, such as the modified SHIRPA ( Figure 4 , Figure 5 ),footprint( Figure 6 , Figure 7 ) and Rod Performance Analysis ( Figure 5 C), similar improvement results were observed.
example 2
[0111] EXAMPLE 2 Weight Loss Stopped by Treatment of the Invention with No Side Effects on Organs and Tissues
[0112] QPSCs also stopped weight loss in SCA3 during disease progression ( image 3 ). Nevertheless, 3 doses of QPSC did not affect the complete blood count (Table 1) or blood biochemical (Table 2) profile in SCA3 individuals. Tables 1 and 2 show that the complete blood count / biochemical profile of 25- to 30-week-old wild-type mice was not different between three doses of QPSCs and saline (three doses administered at one-week intervals). Histopathological analysis showed normal findings in various vital organ tissues after injection of three doses of QPSCs ( figure 2 ).
[0113] Table 1
[0114]
[0115] Data are presented as mean ± SD
[0116] Table 2
[0117]
[0118] Data are presented as mean ± SD
example 3
[0119] Example 3 Immunomodulation and anti-ROS ability and expression of multiple neurotrophic factors and growth factors in mice treated with the present invention
[0120] In vitro studies have shown that QPSC not only has immune regulation and anti-ROS ability ( Figure 10 ), and also has the ability to express multiple neurotrophic factors and growth factors ( Figure 12 ). In vivo studies showed that after QPSC treatment, SCA mice showed improved performance of the rotator under oxidative stress ( Figure 11 ). In addition, QPSC can also be used in vitro ( Figure 13 ) and in vivo ( Figure 14 ) to prevent neuronal loss. Although questions have been raised regarding the possibility of cell migration across the blood-brain barrier (BBB), the ability of QPSCs to localize intracranially via intravenous infusion has been shown ( Figure 8 and Figure 9 ).
[0121] Therefore, it is reasonable to conclude that via intravenous infusion, QPSCs can reach the cerebellum thr...
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