Analysis of the Pathophysiological Mechanism of Bone Lesion in Mucopolysaccharidosis Mouse Model ; Its Natural History and Therapeutic Effect

2016 Fiscal Year Final Research Report

• Project/Area Number: 26461533
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Jikei University School of Medicine
• Principal Investigator: Ida Hiroyuki 東京慈恵会医科大学, 医学部, 教授 (90167255)
• Research Collaborator: MAEDA Kazuhiro 東京慈恵会医科大学, 整形外科学講座, 助教 (50548849) ; WADA Miho 東京慈恵会医科大学, 小児科学講座, 大学院生
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: ムコ多糖症Ⅱ型 / 骨病変 / 病理 / 遺伝子治療

Outline of Final Research Achievements

In this study, we investigate the pathophysiological mechanism of bone lesion in Mucopolysaccharidosis type II (MPS II) , and study the effect of ex vivo gene therapy targeting for hematopoietic stem cells.
At first, we investigated the pathology of bone disorder in MPS II mouse model to detect items as control for evaluating therapeutic effects, and searched for bio-markers reflecting the progress of bone lesion. As a result, in the pathological study the balance between bone formation and resorption has been inclined to bone formation in this model, but we could not detect the efficient bio-marker of mRNA reflecting bone formation and resorption for evaluating the therapeutic effect. We are currently evaluating the effect of ex vivo gene therapy for MPS II mouse model using recombinant lentivirus vector.

Free Research Field

先天代謝異常症