台大研究計畫管理系統

「人類疾病誘導型多潛能幹細胞服務聯盟 (中央研究院轉撥部分經費)」計畫內容

計畫主持人所屬單位	學院醫學院系所醫學系婦產科
計畫主持人	姓名何弘能 email帳號 hnho
經費來源	科技部
計畫編號	106-2319-B-001-003
計畫起迄日	由 106/05/01 至 107/04/30

中文關鍵詞

英文關鍵詞

中文摘要

於2015 年成立的”人類疾病誘導性多潛能幹細胞(iPSC)服務聯盟”為台灣產學研界第一且唯一
之多潛能幹細胞核心設施，提供iPSC 產製核心服務、iPSC 特性檢驗與iPSC 公開寄存服務。
此聯盟成立目的在於協助台灣研究者踏入iPSC 領域，並提供穩定且高品質的服務。此外，聯
盟亦服務使用者利用iPSC 衍生物(如神經、心肌、肝臟細胞)進行藥物開發、前臨床試驗平台
或作為疾病模式研究致病機轉。另外，聯盟致力於教學與訓練，作為台灣生技醫藥與生命科
學研究發展的核心設施平台，來幫助台灣學者進行幹細胞的研究。這樣的服務聯盟也能夠增
加台灣學者研究的國際競爭力，與國際接軌、交流、合作。因此，此核心設施的延續極具有
國內與國際的重要性。
本iPSC 服務聯盟是一個結合眾多專家、多個團隊形成的服務聯盟，主要由中央研究院生物醫
學科學研究所、台灣大學附設醫院、台北榮民總醫院、食品工業發展研究所與國家衛生研究
院五個機構所組成。在過去兩年間，此聯盟已成功產製出15 株對照組iPSC 細胞株、並在生
技醫藥國家型科技計畫經費的補助下服務18 個研究團隊，產製出19 種疾病iPSC(每種疾病三
位病人)，疾病種類包含了帕金森氏症、早發性卵巢衰竭、法布瑞氏症、芳香族L-胺基酸類脫
羧基酶缺乏症、遺傳性精神分裂症、脊髓小腦萎縮症、腓骨肌萎縮症、Leber 遺傳性視神經
萎縮症、單基因突變糖尿病、遺傳性精神分裂症、第一型糖尿病、涎酸酵素缺乏症、少年型
黃斑部病變、乙醛去氫酶基因突變、心房顫動、多囊性腎病、額顳葉失智症、家族性澱粉樣
多發性神經病變、三種罕病(CMT2A, FXS and FSHD)、粒線體突變引發神經性病變等。所有
的疾病iPSC 與對照組iPSC 細胞株皆已或將公開寄存於食品工業發展研究所(FIRDI)的台灣幹
細胞銀行，開放給全台的研究人員申請使用。此外，本聯盟曾於台灣北、中、南、東7 個研
究機構與醫院舉辦服務推廣活動，亦積極參與國內學術年會推廣服務，並不定期舉辦iPSC 教
育訓練課程與新知研討會議。迄今，已成功輔導超過20 個團隊進入iPSC 研究領域。此外，
我們亦協助使用者進行iPSC 後續分化，我們致力於改進各種細胞型式之分化流程，其中包含
了心肌分化、神經分化、肝臟分化、胰島beta 細胞分化、視網膜色素上皮細胞分化、間葉幹
細胞分化、內皮細胞分化與生殖顆粒細胞分化等。
本服務聯盟的每個成員對此計畫皆展現了高度的熱忱，也樂於接受挑戰，解決問題。我們因
此相信，此核心設施若能藉由生技醫藥核心設施平台計畫延續，將有利於台灣產學研界開創
嶄新且令人興奮的研究題目與領域，並對疾病的致病機轉有更深入的瞭解，以作為台灣在新
藥開發、幹細胞治療、以及個人化醫療的研究與發展最堅實的後盾。
關

英文摘要

In 2015, the first and the only Taiwanese induced pluripotent stem cell (iPSC) core facility, named
the Taiwan Human Disease iPSC Service Consortium, was formed to accelerate stem cell research
and development by providing a core service platform for Taiwanese researchers interested in the
iPSC field. Users, whether experienced or inexperienced, can utilize iPSCs as tools for drug
discovery or disease modelling – particularly those who work on the heart diseases, neural
degenerative diseases, and liver diseases - through directed differentiation. The consortium
dedicates a portion of its budget towards education and training, and has become a resource center
for scientists across Taiwan. This will increase the international competitiveness of Taiwanese
research, and so we consider this project to be of both national and international significance.
This iPSC consortium is a group effort, bringing together many experts from across Taiwan to
collaborate on this project. The consortium currently consists of 5 different institutes: IBMS-AS,
NTUH, TVGH, FIRDI, and NHRI. In the past 2 years, we have successfully generated 15 iPSC
lines from the normal Taiwanese population, which will serve as controls, and disease iPSC lines
from more than 19 diseases with 3 patients for each disease type. These disease types include
Parkinson`s disease, premature ovarian failure, Fabry disease, aromatic L-amino acid decarboxylase
deficiency, inherited schizophrenia, spinocerebellar ataxia, Charcot-Marie-Tooth disease, Leber`s
hereditary optic neuropathy, monogenic diabetes, schizophrenia, type 1 diabetes, sialidosis, juvenile
macular degeneration, ALDH2 gene mutation, atrial fibrillation, polycystic kidney disease,
frontotemporal dementia, familial amyloid neuropathy, orphan diseases, and mitochondrial
disease-related neural defects. These disease iPSC lines and normal iPSCs have been, or will be,
banked and publically deposited at FIRDI, available to the original users, and other researchers, in
Taiwan. Moreover, we have held 7 service promotion sessions in different institutes, and attended
several national conferences to promote our service. In addition, for educational purposes, we had
hold several iPSC training courses and symposiums, and had assisted our users to work on iPSC
and further iPSC differentiation. To facilitate cooperation and better assist clinical users in
developing their own research, we have focused on establishing robust differentiation protocols and
characterization for somatic cells of great interest, including cardiomyocytes, neurons, hepatocytes,
pancreatic beta-cells, retinal pigment epithelium cells, mesenchymal stem cells, endothelial cells
and germline-related granulosa cells.
All of the members of this consortium are extremely passionate about this project, and we all
believe that this iPSC consortium represents a huge opportunity for scientific research in Taiwan.
We believe that it will lead to new and exciting research, better understanding of disease
mechanisms, novel drug discovery, personalized medicine and improved outcomes for patients--
which is of course the fundamental goal behind all biomedical research.