Treatment of Morquio A Disease: Navigating Barriers and Advancing Therapies
学友会セミナー
開催情報
| 開催日時 | 2025 年 9 月 1日（月）15:00 ~ 16:00 |
|---|---|
| 開催場所 | 1号館講堂 |
| 講師 | Professor Adriana Montano |
| 所属・職名 | Vice Dean for Research, Dr. Robert Wilmott IMMUNO Chair in Pediatric Research, Department of Pediatrics, Department of Biochemistry and Molecular Biology, School of Medicine, Saint Louis University, St. Louis, MO, USA |
| 演題 | Treatment of Morquio A Disease: Navigating Barriers and Advancing Therapies |
| 世話人 | 〇主たる世話人：Cevayir COBAN（マラリア免疫学分野/ 国際ワクチンデザインセンター） 世話人：古賀 道子 (感染免疫内科) |
概要
Mucopolysaccharidosis type IVA (MPS IVA; Morquio A) is caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase (GALNS), leading to abnormal lysosomal storage of two glycosaminoglycans (GAGs): keratan sulfate and chondroitin-6-sulfate. Current treatments, such as enzyme replacement therapy (ERT) and bone marrow transplantation, are either highly invasive or prohibitively expensive. While ERT remains the gold standard, it has significant limitations, including short-term effects that require weekly administration, which greatly impacts patients' quality of life. This presentation will address the primary challenges associated with ERT, including immune responses, limited efficacy in certain tissues, and high costs. Furthermore, we will explore potential solutions to these challenges, such as gene therapy, enhanced enzyme replacement therapy, and oral tolerance strategies.