PI：浅井 章博
小児の遺伝性肝疾患に対する新しい治療法の開発を目指す浅井研究所へようこそ。私たちは、進行性家族性肝内胆汁うっ滞症やグリコーゲン貯蔵病など、特定の遺伝的疾患に焦点を当てています。これらの病気は、子どもの肝臓に生まれた時から損傷を引き起こし、現在は肝移植が唯一の治療法です。私たちは患者由来のヒト誘導多能性幹細胞（iPS細胞）を用いて、これらの肝疾患のより精密なモデルを作成する技術を開発しました。この技術により、疾患の発症メカニズムを細かく解析し、治療の新たな道を探ることが可能になります。また、iPS細胞の遺伝子編集により、肝疾患に関連する遺伝子を特定し、治療の標的となる可能性を探っています。さらに、mRNA治療法の研究により、遺伝的欠陥を持つ患者さんに新しい治療法を提供できるように研究を進めています。これらの先進的な研究を通じて、小児肝疾患の治療法を革新し、患者さんの健康と将来に貢献することを目指しています。
Welcome to our leading-edge research program, committed to the advancement of therapeutic strategies for pediatric genetic liver diseases, encompassing conditions such as progressive familial intrahepatic cholestasis, glycogen storage disease, and other hepatic disorders with a genetic basis. These disorders lead to hepatic injury and fibrosis from an early developmental stage, necessitating liver transplantation as the prevailing treatment modality.
In our quest for novel therapeutic options, we have been at the vanguard of employing patient-derived human induced pluripotent stem cells (iPSCs). We have developed innovative methods that replicate the embryonic liver environment, promoting the differentiation of iPSCs into hepatic cells within a bi-chamber culture system equipped with a semipermeable membrane. This technique allows for unprecedented fidelity in modeling human liver disease phenotypes, surpassing the capabilities of conventional rodent models.
Additionally, we have refined our genome editing capabilities to execute precise gene knockouts in iPSCs linked to hepatobiliary diseases. This technique affords us an in-depth understanding of the pathophysiological mechanisms that contribute to cholestatic liver injury during the course of human liver development, a previously intractable research area.
Our ongoing efforts are directed toward delineating the molecular pathways that result in hepatocyte damage, with the aim to uncover new points of therapeutic intervention. This could lead to strategies that prevent or halt the advance of liver disease in young patients.
In a significant stride forward, our laboratory has developed platforms for testing mRNA therapeutics in patient-derived stem cells and mature hepatocytes. These groundbreaking findings suggest the feasibility of supplementing mRNA to patients with genetic deficiencies, potentially circumventing the progression to liver failure. Our commitment is unwavering in the pursuit of these scientific endeavors to improve pediatric hepatic healthcare outcomes.
