Neuronal properties, in vivo effects, and pathology of a Huntington's disease patient-derived induced pluripotent stem cells

Stem Cells

Volumn 30, Issue 9, 2012, Pages 2054-2062

  Jeon, Iksoo ^a   Lee, Nayeon ^a   Li, Jia Yi ^b   Park, In Hyun ^c,d   Park, Kyoung Sun ^e   Moon, Jisook ^a   Shim, Sung Han ^a   Choi, Chunggab ^a   Chang, Da Jeong ^a   Kwon, Jihye ^a   Oh, Seung Hun ^a   Shin, Dong Ah ^h   Kim, Hyun Sook ^a   Do, Jeong Tae ^a   Lee, Dong Ryul ^a   Kim, Manho ^f   Kang, Kyung Sun ^f   Daley, George Q ^c   Brundin, Patrik ^b,g   Song, Jihwan ^a  

^a CHA University   (South Korea)

^b LUND UNIVERSITY   (Sweden)

^c HARVARD MEDICAL SCHOOL   (United States)

^d YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e Pohang University of Science and Technology (POSTECH)   (South Korea)

^f Seoul National University   (South Korea)

^g Yonsei University College of Medicine   (South Korea)

^h VAN ANDEL RESEARCH INSTITUTE   (United States)

Author keywords

Aggregate formation; Behavioral recovery; GABAergic neurons; Huntington's disease; Induced pluripotent stem cells; Quinolinic acid

Indexed keywords

4 AMINOBUTYRIC ACID RECEPTOR; BENZYLOXYCARBONYLLEUCYLLEUCYLLEUCINAL; PROTEASOME;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL TRANSPLANTATION; CONTROLLED STUDY; EMBRYONIC STEM CELL; ENZYME INHIBITION; HUMAN; HUMAN CELL; HUNTINGTON CHOREA; MALE; NONHUMAN; PATHOLOGY; PHENOTYPE; PLURIPOTENT STEM CELL; RAT;

ANIMALS; CELL DIFFERENTIATION; DISEASE MODELS, ANIMAL; HUMANS; HUNTINGTON DISEASE; IMMUNOHISTOCHEMISTRY; INDUCED PLURIPOTENT STEM CELLS; MALE; NEURONS; RATS; RATS, SPRAGUE-DAWLEY; STEM CELL TRANSPLANTATION;

EID: 84864601969     PISSN: 10665099     EISSN: None     Source Type: Journal    
DOI: 10.1002/stem.1135     Document Type: Article

References (27)

1. Kelly CM, Dunnett SB, Rosser AE. Medium spiny neurons for transplantation in Huntington's disease. Biochem Soc Trans 2009;37:323-328.
2. Bachoud-Lévi AC, Rémy P, Nguyen JP et al. Motor and cognitive improvements in patients with Huntington's disease after neural transplantation. Lancet 2000;356:1975-1979.
3. Rosser AE, Barker RA, Harrower T et al. Unilateral transplantation of human primary fetal tissue in four patients with Huntington's disease: NEST-UK safety report ISRCTN no 36485475. J Neurol Neurosurg Psychiatry 2002;73:678-685.
4. Gallina P, Paganini M, Lombardini L et al. Human striatal neuroblasts develop and build a striatal-like structure into the brain of Huntington's disease patients after transplantation. Exp Neurol 2010;222:30-41.
5. Björklund A, Lindvall O. Cell replacement therapies for central nervous system disorders. Nat Neurosci 2000;3:537-544.
6. Dunnett SB, Rosser AE. Stem cell transplantation for Huntington's disease. Exp Neurol 2007;203:279-292.
7. Park IH, Arora N, Huo H et al. Disease-specific induced pluripotent stem cells. Cell 2008;134:877-886.
8. Park IH, Lerou PH, Zhao R et al. Generation of human-induced pluripotent stem cells. Nat Protoc 2008;3:1180-1186.
9. Kawasaki H, Mizuseki K, Nishikawa S et al. Induction of midbrain dopaminergic neurons from ES cells by stromal cell-derived inducing activity. Neuron 2000;28:31-40.
10. Kirik D, Rosenblad C, Björklund A. Characterization of behavioral and neurodegenerative changes following partial lesions of the nigrostriatal dopamine system induced by intrastriatal 6-hydroxydopamine in the rat. Exp Neurol 1998;152:259-277.
11. Montoya CP, Campbell-Hope LJ, Pemberton KD et al. The "staircase test": A measure of independent forelimb reaching and grasping abilities in rats. J Neurosci Methods 1991;36:219-228.
12. Ungerstedt U, Arbuthnott GW. Quantitative recording of rotational behavior in rats after 6-hydroxydopamine lesions of the nigrostriatal dopamine system. Brain Res 1970;24:485-493.
13. Yan Y, Yang D, Zarnowska ED et al. Directed differentiation of dopaminergic neuronal subtypes from human embryonic stem cells. Stem Cells 2005;23:781-790.
14. Mukhida K, Hong M, Miles GB et al. A multitarget basal ganglia dopaminergic and GABAergic transplantation strategy enhances behavioural recovery in parkinsonian rats. Brain 2008;131:2106-2126.
15. Kim H, Lee G, Ganat Y et al. miR-371-3 expression predicts neural differentiation propensity in human pluripotent stem cells. Cell Stem Cell 2011;8:695-706.
16. Aubry L, Bugi A, Lefort N et al. Striatal progenitors derived from human ES cells mature into DARPP32 neurons in vitro and in quinolinic acid-lesioned rats. Proc Natl Acad Sci USA 2008;105:16707-16712.
17. Ma L, Hu B, Liu et al. Human embryonic stem cell-derived GABA neurons correct locomotion deficits in quinolinic acid lesioned mice. Cell Stem Cell 2012:10:1-10.
18. Nakao N, Grasbon-Frodl EM, Widner H et al. DARPP-32-rich zones in grafts of lateral ganglionic eminence govern the extent of functional recovery in skilled paw reaching in an animal model of Huntington's disease. Neuroscience 1996;74:959-970.
19. Fricker RA, Barker RA, Fawcett JW et al. A comparative study of preparation techniques for improving the viability of striatal grafts using vital stains, in vitro cultures, and in vivo grafts. Cell Transplant 1996;5:599-611.
20. Li XJ, Li SH, Sharp AH et al. A huntingtin-associated protein enriched in brain with implications for pathology. Nature 1995;378:398-402.
21. Arrasate M, Finkbeiner S. Protein aggregates in Huntington's disease. Exp Neurol 2011 [Epub ahead of print].
22. Zhang N, An MC, Montoro D et al. Characterization of human Huntington's disease cell model from induced pluripotent stem cells. PLoS Curr 2010;2:RRN1193.
23. Camnasio S, Carri AD, Lombardo A et al. The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity. Neurobiol Dis 2012;46:41-51.
24. Zou J, Maeder ML, Mali P et al. Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells. Cell Stem Cell 2009;5:97-110.
25. Benraiss A, Goldman SA. Cellular therapy and induced neuronal replacement for Huntington's disease. 2011;8:77-590.
26. Olson SD, Kambal A, Pollock K et al. Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for reduction of huntingtin. Cell Mol Neurosci 2012;49:271-281.
27. Olson SD, Pollock K, Kambal A et al. Genetically engineered mesenchymal stem cells as a proposed therapeutic for Huntington's disease. Mol Neurobiol 2012:45;87-98.