Lentivector Transduction Improves Outcomes Over Transplantation of Human HSCs Alone in NOD/SCID/Fabry Mice

Fabry disease is a lysosomal storage disorder caused by a deficiency of a-galactosidase A (a-gal A) activity that results in progressive globotriaosylceramide (Gb(3)) deposition. We created a fully congenic nonobese diabetic (NOD)/severe combined immunodeficiency (SCID)/Fabry murine line to facilitate the in vivo assessment of human cell-directed therapies for Fabry disease. This pure line was generated after 11 generations of backcrosses and was found, as expected, to have a reduced immune compartment and background a-gal A activity. Next, we transplanted normal human CD34(+) cells transduced with a control (lentiviral vector-enhanced green fluorescent protein (LV-eGFP)) or a therapeutic bicistronic LV (LV-a-gal A/internal ribosome entry site (IRES)/hCD25). While both experimental groups showed similar engraftment levels, only the therapeutic group displayed a significant increase in plasma a-gal A activity. Gb(3) quantification at 12 weeks revealed metabolic correction in the spleen, lung, and liver for both groups. Importantly, only in the therapeutically-transduced cohort was a significant Gb(3) reduction found in the heart and kidney, key target organs for the amelioration of Fabry disease in humans.Fil: Pacienza, Natalia Alejandra. University Health Network; Canadá. Consejo Nacional de Investigaciones Científicas y Técnicas; ArgentinaFil: Yoshimitsu, Makoto. Kagoshima University; Japón. University Health Network; CanadáFil: Mizue, Nobuo. University Health Network; CanadáFil: Au, Bryan C. Y.. University Health Network; CanadáFil: Wang, James C. M.. University Health Network; CanadáFil: Fan, Xin. University Health Network; CanadáFil: Takenaka, Toshihiro. Kagoshima University; JapónFil: Medin, Jeffrey A. University Health Network; Canadá. University of Toronto; Canad

Congenital Dysfibrinogenemia Presented with Massive Hematomas Formed after Hysterectomy

ArticleCase Reports in Clinical Medicine. 10: 108-116 (2021)journal articl

Unit plan "Considering the United Nations" : A study on the development of a junior high school civics sourse (2)

The purpose in this paper is to develop the lesson plan for teaching social studies in junior high schools to consider the international society. We developed the unit plan “Considering the United Nations." The aim of this plan is to recognize the perspectives and views of the UN. We have prepared two views of society in the international level to plan the unit. One is an idealistic view that there must be no conflict in the international society. Another is a realistic view that in the international society people manage the conflict politically. As students have learned an idealistic view in elementary social studies lessons, we have planed the lesson that they recognize an idealistic view and a realistic view. As a result of this plan, students will be able to consider how to make the international society using two views

Carbonyl Reductase 3 (CBR3) Mediates 9-cis-Retinoic Acid-Induced Cytostatis and is a Potential Prognostic Marker for Oral Malignancy

The molecular mechanisms of growth suppression by retinoic acid (RA) were examined. Our results suggest that the cytostatic effects of RA could be mediated by the activation of endogenous CBR3 gene in oral squamous cell carcinomas (OSCCs), and the expression is a potential marker for oral malignancy

The Structure of Sequence about the Formation of Teaching Contents of History : the case of for primary and secondary history textbooks which Schofield & Sims LTD. published in the 1970s

The aim of this study is to explain the structure of sequence about the formation of teaching contents of history textbooks which attached importance to the modern world. Therefore I used history textbooks which have been published in nearly same times from same publisher, inquired the formation, and the scope and sequence of teaching contents, compared the one for primary education with the one for secondary education, and analyzed the structure of the formation of teaching contents. Consequently, I explicated the scope structure that alternate from the study of change in social life to the study of change in social structure, the sequence structure that advance steadily about social image and the formation of teaching contents of history which gradually acquired ideas which explain the general society

The Formation of Teaching Contents for Making Social Recognition Historically : the case of textbooks published for "Environmental Studies"

The aim of this study is to explain the structure of scope and sequence about the formation of teaching contents of textbooks which make much of shaping social recognition historically, and to show the historical significance of this structure. Therefore I used the plural textbooks which have been published in nearly same time from same publisher for different age's students, inquired the formation and the scope and sequence of teaching contents, and analyzed the structure of the formation of teaching contents.Consequently, I explained the unique structure of scope and sequence concretely in order to make social recognition historically