Using iPS Technology in Biomedical Research

iPS Technology


Scientists have used iPS cell technology to create nerve cells, allowing them to study a range of neurological disorders, including Alzheimer's, Parkinson's, and Huntington's disease, and develop better models for evaluating potential treatments. 1,2,3,4,5,6,7,8,9

  1. Ring KL, Tong LM, Balestra ME, Javier R, Andrews-Zwilling Y, Li G, Walker D, Zhang WR, Kreitzer AC, Huang Y. Direct reprogramming of mouse and human fibroblasts into multipotent neural stem cells with a single factor. Cell Stem Cell. 2012, 11:100–109, Epub 2012 Jun 7.
  2. Almeida S, Zhang Z, Coppola G, Mao W, Futai K, Karydas A, Geschwind MD, Tartaglia MC, Gao F, Gianni D, Sena-Esteves M, Geschwind DH, Miller BL, Farese RV Jr, Gao FB. Induced pluripotent stem cell models of progranulin-deficient frontotemporal dementia uncover specific reversible neuronal defects. Cell Rep. 2012, 2:789–798, Epub 2012 Oct 11.
  3. Dimos J, Rodolfa K, Niakan K, Weisenthal L, Mitsumoto H, Chung W, Croft G, Saphier G, Leibel R, Goland R, Wichterle H, Henderson C, Eggan K. Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science, 2008, 321:1218–1221, Epub 2008 Jul 31.
  4. Soldner F, Hockemeyer D, Beard C, Gao Q, Bell GW, Cook EG, Hargus G, Blak A, Cooper O, Mitalipova M, Isacson O, Jaenisch R. Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell. 2009 136:964–977.
  5. Wernig M, Zhao JP, Pruszak J, Hedlund E, Fu D, Soldner F, Broccoli V, Constantine-Paton M, Isacson O, Jaenisch R. Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease. Proc Natl Acad Sci U S A. 2008, 105:5856–5861. Epub 2008 Apr 7.
  6. HD iPSC Consortium. Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes. Cell Stem Cell, 2012, 11:264–278.
  7. Ebert AD, Yu J, Rose FF Jr, Mattis VB, Lorson CL, Thomson JA, Svendsen CN. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature. 2009, 457:277–280. Epub 2008 Dec 21.
  8. Polentes J, Jendelova P, Cailleret M, Braun H, Romanyuk N, Tropel P, Brenot M, Itier V, Seminatore C, Baldauf K, Turnovcova K, Jirak D, Teletin M, Côme J, Tournois J, Reymann K, Sykova E, Viville S, Onteniente B. Human induced pluripotent stem cells improve stroke outcome and reduce secondary degeneration in the recipient brain. Cell Transplant. 2012 Aug 10. In press.
  9. Chang DJ, Lee N, Park IH, Choi C, Jeon I, Kwon J, Oh SH, Shin DA, Do JT, Lee DR, Lee H, Moon H, Hong KS, Daley GQ, Song J. Therapeutic potential of human induced pluripotent stem cells in experimental stroke. Cell Transplant. 2012 Oct 3. In press.


Scientists have generated iPS-based retinal cells, allowing them to study the causes and potential treatments of a range of diseases that can cause blindness, such as glaucoma and age-related macular degeneration.10,11

  1. Parameswaran S, Balasubramanian S, Babai N, Qiu F, Eudy JD, Thoreson WB, Ahmad I. Induced pluripotent stem cells generate both retinal ganglion cells and photoreceptors: therapeutic implications in degenerative changes in glaucoma and age-related macular degeneration. Stem Cells. 2010, 28:695–703.
  2. Jin Z, Okamoto S, Osakada F, Homma K, Assawachananont J, Hirami Y, Iwata T, Takahashi M. Modeling retinal degeneration using patient-specific induced pluripotent stem cells. PLoS On. 2011 Feb 10;6(2):e17084.


In animal models of spinal cord injuries, scientists implanted iPS cells that then differentiated into functional nerve cells, suggesting a promising cell-replacement therapy that may one day be used to treat spinal cord injuries in people.12

  1. Tsuji O, Miura K, Okada Y, Fujiyoshi K, Mukaino M, Nagoshi N, Kitamura K, Kumagai G, Nishino M, Tomisato S, Higashi H, Nagai T, Katoh H, Kohda K, Matsuzaki Y, Yuzaki M, Ikeda E, Toyama Y, Nakamura M, Yamanaka S, Okano H. Therapeutic potential of appropriately evaluated safe-induced pluripotent stem cells for spinal cord injury. Proc Natl Acad Sci. USA, 2010, 107:12704–12709, Epub 6 Jul 2010


iPS cell technology provides an entirely new platform for understanding the causes of heart disease from congenital heart defects in babies to heart failure in the elderly. This technology offers new tools for evaluating drug treatments and the possibility of restoring heart muscle damaged by heart attack.13,14,15

  1. Lahti AL, Kujala VJ, Chapman H, Koivisto AP, Pekkanen-Mattila M, Kerkelä E, Hyttinen J, Kontula K, Swan H, Conklin BR, Yamanaka S, Silvennoinen O, Aalto-Setälä K. Modeling for long QT syndrome type 2 using human iPS cells demonstrates arrythmogenic characteristics in culture. Dis Model Mech. 2012, 5:220–230.
  2. Efe JA, Hilcove S, Kim J, Zhou H, Ouyang K, Wang G, Chen J, Ding S. Conversion of mouse fibroblasts into cardiomyocytes using a direct reprogramming strategy. Nature 2012, 485, 593–598
  3. Qian L, Huang Y, Spencer CI, Foley A, Vedantham V, Liu L, Conway SJ, Fu JD, Srivastava D. In vivo reprogramming of murine cardiac fibroblasts into induced cardiomyocytes. Nature. 2012, 485:593–598.


Kidney cells created from human iPS cells are helping scientists to better understand kidney disease and to evaluate potential alternative treatments in the hopes of one day eliminating the need for dialysis or transplants.16

  1. Song B, Smink A, Jones C, Callaghan J, Firth S, Bernard C, Laslett A, Kerr P, Ricardo S. The directed differentiation of human iPS cells into kidney podocytes. PLoS ONE 7(9): e46453.


Scientists have generated iPS cell-derived liver cells from patients with liver disease to study its underlying causes and evaluate potential drugs to treat the disease.17

  1. Rashid ST, Corbineau S, Hannan N, Marciniak S, Miranda E, Alexander G, Huang-Doran I, Griffin J, Ahrlund-Richter L, Skepper J, Semple R, Weber A, Lomas DA, Vallier L. Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells. J Clin Invest. 2010, 120: 3127–3136, Epub: 25 Aug 2010.


Researchers have transformed human iPS cells into insulin-producing cells that, when transplanted into mice with Type I diabetes, secrete insulin and normalize blood sugar levels. Such therapies may one day replace insulin injections for people with diabetes. 18

  1. Jeon K, Lim H, Kim J, Thuan NV, Park SH, Lim Y, Choi H, Lee E, Kim J, Lee M, Cho S. Differentiation and transplantation of functional pancreatic beta cells generated from induced pluripotent stem cells derived from a type 1 diabetes mouse model. Stem Cells and Development. 2012, 21:2642–2655


Scientists have converted human skin cells into blood cells through direct reprogramming, bypassing the iPS cell stage altogether. This approach represents the new frontier of cellular reprogramming for disease research.19

  1. Szabo E, Rampalli S, Risueno R, Schnerch A, Mitchell R, Fiebig-Comyn A, Levadoux-Martin M, Bhatia M. Direct conversion of human fibroblasts to multilineage blood progenitors. Nature 2010. 468:521–526.


Scientists have reprogrammed human iPS cells into bone and cartilage cells that may one day be used to treat diseases, such as osteoporosis, and replace damaged or injured cartilage.20,21,22

  1. Jin G, Kim T, Kim J, Won J, Yoo S, Choi S, Kim H. Bone tissue engineering of induced pluripotent stem cells cultured with macrochanneled polymer scaffold. J Biomed Mater Res Part A, 10.1002/jbm.a.34425
  2. Hiramatsu K, Sasagawa S, Outani H, Nakagawa K, Yoshikawa H, Tsumaki N. Generation of hyaline cartilaginous tissue from mouse adult dermal fibroblast culture by defined factors. J Clin Invest. 2011 121: 640–657, Epub: 10 Jan 2011.
  3. Medvedev SP, Grigor'eva EV, Shevchenko AI, Malakhova AA, Dementyeva EV, Shilov AA, Pokushalov EA, Zaidman AM, Aleksandrova MA, Plotnikov EY, Sukhikh GT, Zakian SM. Human induced pluripotent stem cells derived from fetal neural stem cells successfully undergo directed differentiation into cartilage. Stem Cells Devel. 2011, 20: 1099–1112, Epub 17 Oct 2010.