美国研究人员8月7日宣布，利用患者体细胞培育出10种可用于观察遗传疾病在细胞内发展的干细胞。这一进展将有利于寻找这些疾病的治疗方法。
哈佛大学干细胞研究所博士乔治·戴利和同事利用患者皮肤细胞和骨髓，培育出这些干细胞。这些患者罹患疾病包括帕金森氏症、亨廷顿舞蹈病和唐氏综合症等。
这种新技术原理是将患者的普通细胞转化成具有胚胎干细胞特征的干细胞。胚胎干细胞可分化为不同种类的体细胞，进而成长为人体各种组织，例如心脏和大脑组织。这一研究成果发表在美国《细胞》（Cell）杂志网络版上。
干细胞研究所所长道格·梅尔顿说，这些干细胞将有助于研究人员观察这些疾病在人体细胞内的发展情况，“我认为，在数年内我们就会看到这为治疗疾病开启新的大门”。
梅尔顿说，研究所建立了一所实验室，专门储存这些人造干细胞，以加速这方面研究。
人体体细胞“转化”为干细胞曾有先例。美国和日本研究人员2007年11月报告说，他们利用重新编排基因技术，把人体皮肤细胞改造成皮肤干细胞，功能可媲美胚胎干细胞。上星期，哈佛大学另一研究小组说，他们把两名肌肉萎缩性侧索硬化症老年患者的皮肤细胞转化成干细胞，再把这些干细胞培育成神经细胞。
原始出处：
Cell，In press，In-Hyun Park, George Q. Daley
Disease-Specific Induced Pluripotent Stem Cells
In-Hyun Park,1,7 Natasha Arora,1,7 Hongguang Huo,1,7 Nimet Maherali,2,3,7 Tim Ahfeldt,2,5,7 Akiko Shimamura,4 M. William Lensch,1,7,9 Chad Cowan,2,6,7 Konrad Hochedlinger,2,7 and George Q. Daley1,7,8,9,
1 Department of Medicine, Division of Pediatric Hematology Oncology, Children's Hospital Boston, and Dana-Farber Cancer Institute; Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Karp Family Research Building 7214, 300 Longwood Avenue, Boston, MA 02115
2 Massachusetts General Hospital Cancer Center and Center for Regenerative Medicine and Department of Stem Cell and Regenerative Biology, 185 Cambridge Street, Boston, MA 02114, USA
3 Department of Molecular and Cellular Biology, Harvard University, 7 Divinity Avenue, Cambridge, MA 02138, USA
4 Department of Pediatrics, Division of Hematology/Oncology, University of Washington and Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N., Seattle, WA 98109, USA
5 Department of Biochemistry and Molecular Biology II: Molecular Cell Biology, University Medical Center Hamburg-Eppendorf, Hamburg 20246, Germany
6 Stowers Medical Institute, Karp Family Research Building 7214, 300 Longwood Avenue, Boston, MA 02115
7 Harvard Stem Cell Institute, Karp Family Research Building 7214, 300 Longwood Avenue, Boston, MA 02115
8 Division of Hematology, Brigham and Women's Hospital, Karp Family Research Building 7214, 300 Longwood Avenue, Boston, MA 02115
9 Howard Hughes Medical Institute, Children's Hospital Boston, Karp Family Research Building 7214, 300 Longwood Avenue, Boston, MA 02115
Corresponding author
George Q. Daley
george.daley@childrens.harvard.edu
Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance; these diseases include adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. Such disease-specific stem cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation and drug development.
