E-ISSN 2146-8362
 

Review Article 


Gene Therapy and Stem Cells in the Treatment of Congenital Diseases

Paula Rubya Souza Camara.

Abstract
Several congenital diseases are particularly attractive candidates for intervention using gene therapy since the underlying molecular bases for most of the monogenic disorders are well-understood. Transplantation of ex vivo genetically modified stem cells has also shown promise. Although all of these systems are meritorious and worthy of continued investigation, this mini-review article focused on the platforms that have received the most attention and that are maturing in the clinical setting; in particular, the potential of in vivo gene therapy and human-induced pluripotent stem cells. Studies of apparently disparate diseases that are presumably linked through shared metabolic pathways are likely to provide greater insights into the biology of the diseases. This and other opportunities for exchange will hopefully foster acceleration in the development of new and innovative therapies for these devastating diseases.

Key words: congenital diseases, gene therapy, pluripotency, stem cells, frozen embryos


 
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REFERENCES
1. McCain J. The future of gene therapy. Biotechnol Healthc 2005;2:52-60. [Pubmed]    [PMC Free Fulltext]   
2. Malik P, Arumugam PI. Gene Therapy for beta-thalassemia. Hematology Am Soc Hematol Educ Program 2005:45-50. [DOI via Crossref]    [Pubmed]   
3. Löscher W, Gernert M, Heinemann U. Cell and gene therapies in epilepsy-promising avenues or blind alleys? Trends Neurosci 2008;31:62-73. [DOI via Crossref]    [Pubmed]   
4. Simonato M. Gene therapy for epilepsy. Epilepsy Behav 2014;38:125-30. [DOI via Crossref]    [Pubmed]   
5. Macpherson JL, Rasko JE. Clinical potential of gene therapy: towards meeting the demand. Intern Med J 2014;44:224-33. [DOI via Crossref]    [Pubmed]   
6. High KH, Nathwani A, Spencer T, Lillicrap D. Current status of haemophilia gene therapy. Haemophilia 2014;20 Suppl 4:43-9. [DOI via Crossref]    [Pubmed]   
7. Al-Saikhan FI. The gene therapy revolution in ophthalmology. Saudi J Ophthalmol 2013;27:107-11. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
8. Bible E. Sensory systems: Promising results in a gene therapy trial for retinal disease. Nat Rev Neurol 2014;10:123. [DOI via Crossref]    [Pubmed]   
9. MacLaren RE, Groppe M, Barnard AR, Cottriall CL, Tolmachova T, Seymour L, et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet 2014;383:1129-37. [DOI via Crossref]   
10. Barzon L, Bonaguro R, Palù G, Boscaro M. New perspectives for gene therapy in endocrinology. Eur J Endocrinol 2000;143:447-66. [DOI via Crossref]    [Pubmed]   
11. Evans CH, Ghivizzani SC, Kang R, Muzzonigro T, Wasko MC, Herndon JH, et al. Gene therapy for rheumatic diseases. Arthritis Rheum 1999;42:1-16. [DOI via Crossref]   
12. Evans CH, Ghivizzani SC, Robbins PD. Gene therapy of the rheumatic diseases: 1998 to 2008. Arthritis Res Ther 2009;11:209. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
13. Evans CH, Ghivizzani SC, Robbins PD. Arthritis gene therapy and its tortuous path into the clinic. Transl Res 2013;161:205-16. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
14. Iwata N, Ozawa K, Saido TC. Gene therapy for Alzheimer' s disease. Nihon Rinsho 2005;63:394-400. [Pubmed]   
15. Kaiser R, Thiel E, Kreuser ED. Human gene therapy in gastrointestinal diseases: In vivo and in vitro approaches. Recent Results Cancer Res 1996;142:51-61. [DOI via Crossref]    [Pubmed]   
16. Blum HE, Wieland S, von Weizsäcker F. Gene therapy: basic concepts and applications in gastrointestinal diseases. Digestion 1997;58:87-97. [DOI via Crossref]    [Pubmed]   
17. Forbes SJ, Hodgson HJ. Review article: gene therapy in gastroenterology and hepatology. Aliment Pharmacol Ther 1997;11:823-36. [DOI via Crossref]   
18. Gottschalk U, Chan S. Somatic gene therapy. Present situation and future perspective. Arzneimittelforschung 1998;48:1111-20. [Pubmed]   
19. Camilleri M. Enteric nervous system disorders: genetic and molecular insights for the neurogastroenterologist. Neurogastroenterol Motil 2001;13:277-95. [DOI via Crossref]    [Pubmed]   
20. Flotte TR, Laube BL. Gene therapy in cystic fibrosis. Chest 2001;120 3 Suppl:124S-31S. [DOI via Crossref]    [Pubmed]   
21. Prieto J, Herraiz M, Sangro B, Qian C, Mazzolini G, Melero I, et al. The promise of gene therapy in gastrointestinal and liver diseases. Gut 2003;52 Suppl 2:ii49-54. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
22. Prieto J, Qian C, Hernandez-Alcoceba R, Gonzalez-Aseguinolaza G, Mazzolini G, Sangro B, et al. Gene therapy of liver diseases. Expert Opin Biol Ther 2004;4:1073-91. [DOI via Crossref]    [Pubmed]   
23. Qian C, Prieto J. Gene therapy of cancer: Induction of anti-tumor immunity. Cell Mol Immunol 2004;1:105-11. [Pubmed]   
24. Benaim E, Sorrentino BP. Gene therapy in pediatric oncology. Invest New Drugs 1996;14:87-99. [DOI via Crossref]    [Pubmed]   
25. Qian C, Liu XY, Prieto J. Therapy of cancer by cytokines mediated by gene therapy approach. Cell Res 2006;16:182-8. [DOI via Crossref]    [Pubmed]   
26. Barzon L, Stefani AL, Pacenti M, Palù G. Versatility of gene therapy vectors through viruses. Expert Opin Biol Ther 2005;5:639-62. [DOI via Crossref]    [Pubmed]   
27. Keung EZ, Nelson PJ, Conrad C. Concise review: genetically engineered stem cell therapy targeting angiogenesis and tumor stroma in gastrointestinal malignancy. Stem Cells 2013;31:227-35. [DOI via Crossref]    [Pubmed]   
28. Lundberg C, Björklund T, Carlsson T, Jakobsson J, Hantraye P, Déglon N, et al. Applications of lentiviral vectors for biology and gene therapy of neurological disorders. Curr Gene Ther 2008;8:461-73. [DOI via Crossref]    [Pubmed]   
29. Nanou A, Azzouz M. Gene therapy for neurodegenerative diseases based on lentiviral vectors. Prog Brain Res 2009;175:187-200. [DOI via Crossref]   
30. Braverman NE, D'Agostino MD, Maclean GE. Peroxisome biogenesis disorders: Biological, clinical and pathophysiological perspectives. Dev Disabil Res Rev 2013;17:187-96. [DOI via Crossref]    [Pubmed]   
31. Hung KL, Wang JS, Keng WT, Chen HJ, Liang JS, Ngu LH, et al. Mutational analyses on X-linked adrenoleukodystrophy reveal a novel cryptic splicing and three missense mutations in the ABCD1 gene. Pediatr Neurol 2013;49:185-90. [DOI via Crossref]    [Pubmed]   
32. Ezgu F. Recent advances in the molecular diagnosis of inborn errors of metabolism. Clin Biochem 2014;47:759-60. [DOI via Crossref]    [Pubmed]   
33. Cheng SH. Gene therapy for the neurological manifestations in lysosomal storage disorders. J Lipid Res 2014;55:1827-38. [DOI via Crossref]    [Pubmed]   
34. Macnab S, Whitehouse A. Progress and prospects: human artificial chromosomes. Gene Ther 2009;16:1180-8. [DOI via Crossref]    [Pubmed]   
35. Beg S, Rizwan M, Sheikh AM, Hasnain MS, Anwer K, Kohli K. Advancement in carbon nanotubes: basics, biomedical applications and toxicity. J Pharm Pharmacol 2011;63:141-63. [DOI via Crossref]    [Pubmed]   
36. Mallick K, Strydom AM. Biophilic carbon nanotubes. Colloids Surf B Biointerfaces 2013;105:310-8. [DOI via Crossref]    [Pubmed]   
37. Rastogi V, Yadav P, Bhattacharya SS, Mishra AK, Verma N, Verma A, et al. Carbon nanotubes: an emerging drug carrier for targeting cancer cells. J Drug Deliv 2014;2014:670815. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
38. Plusa B, Piotrowska K, Zernicka-Goetz M. Sperm entry position provides a surface marker for the first cleavage plane of the mouse zygote. Genesis 2002;32:193-8. [DOI via Crossref]    [Pubmed]   
39. Takahashi K, Okita K, Nakagawa M, Yamanaka S. Induction of pluripotent stem cells from fibroblast cultures. Nat Protoc 2007;2:3081-9. [DOI via Crossref]    [Pubmed]   
40. Yu J, Vodyanik MA, Smuga-Otto K, Antosiewicz-Bourget J, Frane JL, Tian S, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007;318:1917-20. [DOI via Crossref]    [Pubmed]   
41. Wilmut I. The first direct reprogramming of adult human fibroblasts. Cell Stem Cell 2007;1:593-4. Induction of pluripotency: from mouse to human. Cell 2007;131:834-5.
42. Zaehres H, Schöler HR
43. Wernig M, Zhao JP, Pruszak J, Hedlund E, Fu D, Soldner F, et al. 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;105:5856-61. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
44. Okita K, Nakagawa M, Hyenjong H, Ichisaka T, Yamanaka S. Generation of mouse induced pluripotent stem cells without viral vectors. Science 2008;322:949-53. [DOI via Crossref]    [Pubmed]   
45. Yakubov E, Rechavi G, Rozenblatt S, Givol D. Reprogramming of human fibroblasts to pluripotent stem cells using mRNA of four transcription factors. Biochem Biophys Res Commun 2010;394:189-93. [DOI via Crossref]    [Pubmed]   
46. Pu J, Jiang H, Zhang B, Feng J. Redefining Parkinson's disease research using induced pluripotent stem cells. Curr Neurol Neurosci Rep 2012;12:392-8. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
47. Bishop AE. Pulmonary epithelial stem cells. Cell Prolif 2004;37(1):89-96. [DOI via Crossref]    [Pubmed]   
48. Liu X, Driskell RR, Engelhardt JF. Airway glandular development and stem cells. Curr Top Dev Biol 2004;64:33-56. [DOI via Crossref]   
49. Hoffman JA, Merrill BJ. New and renewed perspectives on embryonic stem cell pluripotency. Front Biosci 2007;12:3321-32. [DOI via Crossref]   
50. Thapar N. New frontiers in the treatment of Hirschsprung disease. J Pediatr Gastroenterol Nutr 2009;48 Suppl 2:S92-4. [DOI via Crossref]    [Pubmed]   
51. Dhaulakhandi DB, Rohilla S, Rattan KN. Neural tube defects: review of experimental evidence on stem cell therapy and newer treatment options. Fetal Diagn Ther 2010;28:72-8. [DOI via Crossref]    [Pubmed]   
52. Roomans GM. Tissue engineering and the use of stem/progenitor cells for airway epithelium repair. Eur Cell Mater 2010;19:284-99. [Pubmed]   
53. Somers A, Jean JC, Sommer CA, Omari A, Ford CC, Mills JA, et al. Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette. Stem Cells 2010;28:1728-40. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
54. Hotta R, Natarajan D, Burns AJ, Thapar N. Stem cells for GI motility disorders. Curr Opin Pharmacol 2011;11:617-23. [DOI via Crossref]    [Pubmed]   
55. Bernstein HS, Srivastava D. Stem cell therapy for cardiac disease. Pediatr Res 2012;71:491-9. [DOI via Crossref]    [Pubmed]   
56. Files MD, Boucek RJ. 'Shovel-Ready' applications of stem cell advances for pediatric heart disease. Curr Opin Pediatr 2012;24:577-83. [DOI via Crossref]    [Pubmed]   
57. Wong AP, Bear CE, Chin S, Pasceri P, Thompson TO, Huan LJ, et al. Directed differentiation of human pluripotent stem cells into mature airway epithelia expressing functional CFTR protein. Nat Biotechnol 2012;30:876-82. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
58. Green MD, Huang SX, Snoeck HW. Stem cells of the respiratory system: from identification to differentiation into functional pithelium. Bioessays 2013;35:261-70. [DOI via Crossref]    [Pubmed]   
59. Maher KO, Xu C. Marching towards regenerative cardiac therapy with human pluripotent stem cells. Discov Med 2013;15:349-56. [Pubmed]    [PMC Free Fulltext]   
60. Moodley Y, Thompson P, Warburton D. Stem cells: a recapitulation of development. Respirology 2013;18:1167-76. [DOI via Crossref]    [Pubmed]   
61. Nakamura K, Hirano K, Wu SM. iPS cell modeling of cardiometabolic diseases. J Cardiovasc Transl Res 2013;6:46-53. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
62. Saadai P, Wang A, Nout YS, Downing TL, Lofberg K, Beattie MS, et al. Human induced pluripotent stem cell-derived neural crest stem cells integrate into the injured spinal cord in the fetal lamb model of myelomeningocele. J Pediatr Surg 2013;48:158-63. [DOI via Crossref]    [Pubmed]   
63. Barad L, Schick R, Zeevi-Levin N, Itskovitz-Eldor J, Binah O. Human embryonic stem cells vs human induced pluripotent stem cells for cardiac repair. Can J Cardiol 2014;30:1279-87. [DOI via Crossref]    [Pubmed]   
64. Chen G, Li S, Karakikes I, Ren L, Chow MZ, Chopra A, et al. Phospholamban as a crucial determinant of the inotropic response of human pluripotent stem cell-derived ventricular cardiomyocytes and engineered 3D tissue constructs. Circ Arrhythm Electrophysiol 2014.
65. Citro L, Naidu S, Hassan F, Kuppusamy ML, Kuppusamy P, Angelos MG, et al. Comparison of human induced pluripotent stem-cell derived cardiomyocytes with human mesenchymal stem cells following acute myocardial infarction. PLoS One 2014;9:e116281. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
66. Dianat N, Dubois-Pot-Schneider H, Steichen C, Desterke C, Leclerc P, Raveux A, et al. Generation of functional cholangiocyte-like cells from human pluripotent stem cells and HepaRG cells. Hepatology 2014;60:700-14. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
67. Doerr L, Thomas U, Guinot DR, Bot CT, Stoelzle-Feix S, Beckler M, et al. New easy-to-use hybrid system for extracellular potential and impedance recordings. J Lab Autom 2014. [Pubmed]   
68. Drawnel FM, Boccardo S, Prummer M, Delobel F, Graff A, Weber M, et al. Disease modeling and phenotypic drug screening for diabetic cardiomyopathy using human induced pluripotent stem cells. Cell Rep 2014;9:810-21. [DOI via Crossref]    [Pubmed]   
69. Egashira T, Yuasa S, Tohyama S, Kuroda Y, Suzuki T, Seki T, et al. Patientspecific induced pluripotent stem cell models: Characterization of iPS cell-derived cardiomyocytes. Methods Mol Biol 2014. [DOI via Crossref]    [Pubmed]   
70. Firth AL, Dargitz CT, Qualls SJ, Menon T, Wright R, Singer O, et al. Generation of multiciliated cells in functional airway epithelia from human induced pluripotent stem cells. Proc Natl Acad Sci U S A 2014;111:E1723-30. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
71. Földes G, Matsa E, Kriston-Vizi J, Leja T, Amisten S, Kolker L, et al. Aberrant a-adrenergic hypertrophic response in cardiomyocytes from human induced pluripotent cells. Stem Cell Reports 2014;3:905-14. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
72. Gomperts BN. Induction of multiciliated cells from induced pluripotent stem cells. Proc Natl Acad Sci U S A 2014;111:6120-1. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
73. Hetz S, Acikgoez A, Voss U, Nieber K, Holland H, Hegewald C, et al. In vivo transplantation of neurosphere-like bodies derived from the human postnatal and adult enteric nervous system: a pilot study. PLoS One 2014;9:e93605. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
74. Holmgren G, Synnergren J, Bogestål Y, Améen C, Åkesson K, Holmgren S, et al. Identification of novel biomarkers for doxorubicininduced toxicity in human cardiomyocytes derived from pluripotent stem cells. Toxicology 2015;328:102-11. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
75. Huang SX, Islam MN, O'Neill J, Hu Z, Yang YG, Chen YW, et al. Efficient generation of lung and airway epithelial cells from human pluripotent stem cells. Nat Biotechnol 2014;32:84-91. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
76. Jiang Y, Habibollah S, Tilgner K, Collin J, Barta T, Al-Aama JY, et al. An induced pluripotent stem cell model of hypoplastic left heart syndrome (HLHS) reveals multiple expression and functional differences in HLHS-derived cardiac myocytes. Stem Cells Transl Med 2014;3:416-23. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
77. Jones DK, Liu F, Vaidyanathan R, Eckhardt LL, Trudeau MC, Robertson GA. hERG 1b is critical for human cardiac repolarization. Proc Natl Acad Sci U S A 2014;111:18073-7. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
78. Kobayashi J, Yoshida M, Tarui S, Hirata M, Nagai Y, Kasahara S, et al. Directed differentiation of patient-specific induced pluripotent stem cells identifies the transcriptional repression and epigenetic modification of NKX2-5, HAND1, and NOTCH1 in hypoplastic left heart syndrome. PLoS One 2014;9:e102796. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
79. Lim SY, Sivakumaran P, Crombie DE, Dusting GJ, Pébay A, Dilley RJ. Enhancing human cardiomyocyte differentiation from induced pluripotent stem cells with trichostatin A. Methods Mol Biol 2014. [DOI via Crossref]   
80. Lu J, Wei H, Wu J, Jamil MF, Tan ML, Adenan MI, et al. Evaluation of the cardiotoxicity of mitragynine and its analogues using human induced pluripotent stem cell-derived cardiomyocytes. PLoS One 2014;9:e115648. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
81. Natarajan D, Cooper J, Choudhury S, Delalande JM, McCann C, Howe SJ, et al. Lentiviral labeling of mouse and human enteric nervous system stem cells for regenerative medicine studies. Neurogastroenterol Motil 2014;26:1513-8. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
82. Pointon A, Harmer AR, Dale IL, Abi-Gerges N, Bowes J, Pollard C, et al. Assessment of cardiomyocyte contraction in human-induced pluripotent stem cell-derived cardiomyocytes. Toxicol Sci 2014. pii: kfu312. [Pubmed]   
83. Ramalingam S, Annaluru N, Kandavelou K, Chandrasegaran S. TALEN-mediated generation and genetic correction of diseasespecific human induced pluripotent stem cells. Curr Gene Ther 2014;14:461-72. [DOI via Crossref]    [Pubmed]   
84. Raval KK, Tao R, White BE, De Lange WJ, Koonce CH, Yu J, et al. Pompe disease results in a golgi-based glycosylation deficit in human induced pluripotent stem cell-derived cardiomyocytes. J Biol Chem 2015;290:3121-36. [DOI via Crossref]    [Pubmed]   
85. Sallam K, Kodo K, Wu JC. Modeling inherited cardiac disorders. Circ J 2014;78:784-94. [DOI via Crossref]    [Pubmed]   
86. Sargent RG, Suzuki S, Gruenert DC. Nuclease-mediated doublestrand break (DSB) enhancement of small fragment homologous recombination (SFHR) gene modification in human-induced pluripotent stem cells (hiPSCs). Methods Mol Biol 2014;1114:279-90. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
87. van den Heuvel NH, van Veen TA, Lim B, Jonsson MK. Lessons from the heart: Mirroring electrophysiological characteristics during cardiac development to in vitro differentiation of stem cell derived cardiomyocytes. J Mol Cell Cardiol 2014;67:12-25. [DOI via Crossref]    [Pubmed]   
88. Wong AP, Rossant J. Generation of Lung Epithelium from Pluripotent Stem Cells. Curr Pathobiol Rep 2013;1:137-45. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
89. Yang X, Pabon L, Murry CE. Engineering adolescence: maturation of human pluripotent stem cell-derived cardiomyocytes. Circ Res 2014;114:511-23. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
90. Ye L, Chang YH, Xiong Q, Zhang P, Zhang L, Somasundaram P, et al. Cardiac repair in a porcine model of acute myocardial infarction with human induced pluripotent stem cell-derived cardiovascular cells. Cell Stem Cell 2014;15:750-61. [DOI via Crossref]    [Pubmed]   
91. Zanella F, Sheikh F. Patient-Specific Induced Pluripotent Stem Cell Models: Generation and Characterization of Cardiac Cells. Methods Mol Biol 2014. [DOI via Crossref]    [Pubmed]   
92. Zarogoulidis P, Hohenforst-Schmidt W, Huang H, Sahpatzidou D, Freitag L, Sakkas L, et al. A gene therapy induced emphysema model and the protective role of stem cells. Diagn Pathol 2014 14;9:195.
93. Zhang J, Qu J, Wang J. Patch clamp apply in cardiomyocytes derived from patient's iPS cells for individual anticancer therapy. Int J Clin Exp Med 2014;7:4475-8. [Pubmed]    [PMC Free Fulltext]   
94. Zhang L, Guo J, Zhang P, Xiong Q, Wu SC, Xia L, et al. Derivation and high engraftment of patient-specific cardiomyocyte sheet using induced pluripotent stem cells generated from adult cardiac fibroblast. Circ Heart Fail 2015;8:156-66. [DOI via Crossref]    [Pubmed]   
95. Zhang M, D'Aniello C, Verkerk AO, Wrobel E, Frank S, Ward-van Oostwaard D, et al. Recessive cardiac phenotypes in induced pluripotent stem cell models of Jervell and Lange-Nielsen syndrome: Disease mechanisms and pharmacological rescue. Proc Natl Acad Sci U S A 2014;111:E5383-92. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
96. Chen LW, Kuang F, Wei LC, Ding YX, Yung KK, Chan YS. Potential application of induced pluripotent stem cells in cell replacement therapy for Parkinson's disease. CNS Neurol Disord Drug Targets 2011;10:449-58. [DOI via Crossref]    [Pubmed]   
97. Nishimura K, Takahashi J. Therapeutic application of stem cell technology toward the treatment of Parkinson's disease. Biol Pharm Bull 2013;36:171-5. [DOI via Crossref]    [Pubmed]   
98. Koba C, Haruta M, Matsunaga Y, Matsumura K, Haga E, Sasaki Y, et al. Therapeutic effect of human iPS-cell-derived myeloid cells expressing IFN-ß against peritoneally disseminated cancer in xenograft models. PLoS One 2013;8:e67567. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
99. Wakao H, Yoshikiyo K, Koshimizu U, Furukawa T, Enomoto K, Matsunaga T, et al. Expansion of functional human mucosalassociated invariant T cells via reprogramming to pluripotency and redifferentiation. Cell Stem Cell 2013;12:546-58. [DOI via Crossref]    [Pubmed]   
100. Musunuru K. Genome editing of human pluripotent stem cells to generate human cellular disease models. Dis Model Mech 2013;6:896-904. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
101. Sundberg M, Bogetofte H, Lawson T, Jansson J, Smith G, Astradsson A, et al. Improved cell therapy protocols for Parkinson's disease based on differentiation efficiency and safety of hESC-, hiPSC-, and non-human primate iPSC-derived dopaminergic neurons. Stem Cells 2013;31:1548-62. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
102. Imaizumi Y, Okano H. Modeling human neurological disorders with induced pluripotent stem cells. J Neurochem 2014;129:388-99. [DOI via Crossref]    [Pubmed]   
103. Makridakis M, Roubelakis MG, Vlahou A. Stem cells: insights into the secretome. Biochim Biophys Acta 2013;1834:2380-4. [DOI via Crossref]    [Pubmed]   
104. Zimmerlin L, Park TS, Zambidis ET, Donnenberg VS, Donnenberg AD. Mesenchymal stem cell secretome and regenerative therapy after cancer. Biochimie 2013;95:2235-45. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
105. Maumus M, Jorgensen C, Noël D. Mesenchymal stem cells in regenerative medicine applied to rheumatic diseases: role of secretome and exosomes. Biochimie 2013;95:2229-34. [DOI via Crossref]    [Pubmed]   
106. Drago D, Cossetti C, Iraci N, Gaude E, Musco G, Bachi A, et al. The stem cell secretome and its role in brain repair. Biochimie 2013;95:2271-85. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
107. Drouet V, Ruiz M, Zala D, Feyeux M, Auregan G, Cambon K, et al. Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells. PLoS One 2014;9:e99341. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
108. De Coppi P, Bartsch G Jr, Siddiqui MM, Xu T, Santos CC, Perin L, et al. Isolation of amniotic stem cell lines with potential for therapy. Nat Biotechnol 2007;25:100-6. [DOI via Crossref]    [Pubmed]   
109. Skardal A, Mack D, Kapetanovic E, Atala A, Jackson JD, Yoo J, et al. Bioprinted amniotic fluid-derived stem cells accelerate healing of large skin wounds. Stem Cells Transl Med 2012;1:792-802. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
110. Pozzobon M, Piccoli M, Schiavo AA, Atala A, De Coppi P. Isolation of c-Kit+ human amniotic fluid stem cells from second trimester. Methods Mol Biol 2013;1035:191-8. [DOI via Crossref]    [Pubmed]   
111. Skardal A, Mack D, Atala A, Soker S. Substrate elasticity controls cell proliferation, surface marker expression and motile phenotype in amniotic fluid-derived stem cells. J Mech Behav Biomed Mater 2013;17:307-16. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
112. Pipino C, Di Tomo P, Mandatori D, Cianci E, Lanuti P, Cutrona MB, et al. Calcium sensing receptor activation by calcimimetic R-568 in human amniotic fluid mesenchymal stem cells: correlation with osteogenic differentiation. Stem Cells Dev 2014;23:2959-71. [DOI via Crossref]    [Pubmed]   
113. Xiao GY, Liu IH, Cheng CC, Chang CC, Lee YH, Cheng WT, et al. Amniotic fluid stem cells prevent follicle atresia and rescue fertility of mice with premature ovarian failure induced by chemotherapy. PLoS One 2014;9:e106538. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
114. Ma X, Li H, Xin S, Ma Y, Ouyang T. Human amniotic fluid stem cells support undifferentiated propagation and pluripotency of human embryonic stem cell without b-FGF in a density dependent manner. Int J Clin Exp Pathol 2014;7:4661-73. [Pubmed]    [PMC Free Fulltext]   
115. Ramachandra DL, Shaw SS, Shangaris P, Loukogeorgakis S, Guillot PV, Coppi PD, David AL. In utero therapy for congenital disorders using amniotic fluid stem cells. Front Pharmacol 2014;5:270. [DOI via Crossref]    [Pubmed]    [PMC Free Fulltext]   
116. Tajiri N, Acosta S, Portillo-Gonzales GS, Aguirre D, Reyes S, Lozano D, et al. Therapeutic outcomes of transplantation of amniotic fluidderived stem cells in experimental ischemic stroke. Front Cell Neurosci 2014 13;8:227.
117. Kastenberg ZJ, Odorico JS. Alternative sources of pluripotency: science, ethics, and stem cells. Transplant Rev (Orlando) 2008;22:215-22. [DOI via Crossref]    [Pubmed]   
118. Kovacs GT, Breheny SA, Dear MJ. Embryo donation at an Australian university in-vitro fertilisation clinic: issues and outcomes. Med J Aust 2003;178:127-9. [Pubmed]   
119. Alizadeh L, Omani Samani R. Using fertile couples as embryo donors: An ethical dilemma. Iran J Reprod Med 2014;12:169-74. [Pubmed]    [PMC Free Fulltext]   
120. Amesse LS, Srivastava G, Uddin D, Pfaff-Amesse T. Comparison of cryopreserved sperm in vaporous and liquid nitrogen. J Reprod Med 2003;48:319-24. [Pubmed]   
121. Crespilho AM, Nichi M, Guasti PN, Freitas-Dell'Aqua CP, Sá Filho MF, Maziero RR, et al. Sperm fertility and viability following 48h of refrigeration: evaluation of different extenders for the preservation of bull semen in liquid state. Anim Reprod Sci 2014;146:126-33. [DOI via Crossref]    [Pubmed]   

How to Cite this Article
Pubmed Style

Paula Rubya Souza Camara. Gene Therapy and Stem Cells in the Treatment of Congenital Diseases. J Interdiscip Histopathol. 2015; 3(2): 58-62. doi:10.5455/jihp.20150128125706


Web Style

Paula Rubya Souza Camara. Gene Therapy and Stem Cells in the Treatment of Congenital Diseases. http://www.ejmjih.com/?mno=1669 [Access: August 14, 2018]. doi:10.5455/jihp.20150128125706


AMA (American Medical Association) Style

Paula Rubya Souza Camara. Gene Therapy and Stem Cells in the Treatment of Congenital Diseases. J Interdiscip Histopathol. 2015; 3(2): 58-62. doi:10.5455/jihp.20150128125706



Vancouver/ICMJE Style

Paula Rubya Souza Camara. Gene Therapy and Stem Cells in the Treatment of Congenital Diseases. J Interdiscip Histopathol. (2015), [cited August 14, 2018]; 3(2): 58-62. doi:10.5455/jihp.20150128125706



Harvard Style

Paula Rubya Souza Camara (2015) Gene Therapy and Stem Cells in the Treatment of Congenital Diseases. J Interdiscip Histopathol, 3 (2), 58-62. doi:10.5455/jihp.20150128125706



Turabian Style

Paula Rubya Souza Camara. 2015. Gene Therapy and Stem Cells in the Treatment of Congenital Diseases. Journal of Interdisciplinary Histopathology, 3 (2), 58-62. doi:10.5455/jihp.20150128125706



Chicago Style

Paula Rubya Souza Camara. "Gene Therapy and Stem Cells in the Treatment of Congenital Diseases." Journal of Interdisciplinary Histopathology 3 (2015), 58-62. doi:10.5455/jihp.20150128125706



MLA (The Modern Language Association) Style

Paula Rubya Souza Camara. "Gene Therapy and Stem Cells in the Treatment of Congenital Diseases." Journal of Interdisciplinary Histopathology 3.2 (2015), 58-62. Print. doi:10.5455/jihp.20150128125706



APA (American Psychological Association) Style

Paula Rubya Souza Camara (2015) Gene Therapy and Stem Cells in the Treatment of Congenital Diseases. Journal of Interdisciplinary Histopathology, 3 (2), 58-62. doi:10.5455/jihp.20150128125706





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