Skip to main content Accessibility help
×
Hostname: page-component-848d4c4894-4hhp2 Total loading time: 0 Render date: 2024-05-18T01:56:53.542Z Has data issue: false hasContentIssue false

19 - Principles for Targeting Adult Tissue Stem Cells to Achieve Durable Gene and Gene Editing Therapies

from Part IV - Genome Editing in Stem Cells and Regenerative Biology

Published online by Cambridge University Press:  30 July 2018

Krishnarao Appasani
Affiliation:
GeneExpression Systems, Inc.
Get access

Summary

Image of the first page of this content. For PDF version, please use the ‘Save PDF’ preceeding this image.'
Type
Chapter
Information
Genome Editing and Engineering
From TALENs, ZFNs and CRISPRs to Molecular Surgery
, pp. 285 - 300
Publisher: Cambridge University Press
Print publication year: 2018

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

Aravalli, RN, Steer, CJ. 2016. Gene editing technology as an approach to the treatment of liver diseases. Expert Opin Biol Ther 16: 595608.CrossRefGoogle Scholar
Arber, N, Zajicek, G. 1990. Streaming liver VI: streaming intra-hepatic bile ducts. Liver 10: 205208.CrossRefGoogle ScholarPubMed
Arber, N, Zajicek, G, Ariel, I. 1988. The streaming liver II. Hepatocyte life history. Liver 8: 8087.CrossRefGoogle ScholarPubMed
Benedetti, A, Jézéquel, AM, Orlandi, F. 1988. Preferential distribution of apoptotic bodies in the acinar zone 3 of normal human and rat liver. J Hepatol 7: 319324.CrossRefGoogle ScholarPubMed
Blikkendaal-Lieftinck, LF, Kooij, M, Kramer, MF, Otter, WD. 1977. Cell kinetics in the liver of rats under normal and abnormal dietary conditions. An autoradiographic study by means of [3H] thymidine. Exp Mol Path 26: 184192.CrossRefGoogle ScholarPubMed
Carroll, J. 2015. Bluebird shares dented after gene therapy patient experiences a setback. FierceBiotech. www.fiercebiotech.com/r-d/bluebird-shares-dented-after-gene-therapy-patient-experiences-a-setback (accessed November 2017).Google Scholar
Chira, S, Jackson, CS, Oprea, I, et al. 2015. Progresses towards safe and efficient gene therapy vectors. Oncotarget 6: 3067530703.CrossRefGoogle ScholarPubMed
Fahrner, J, Labruyere, WT, Gaunitz, C, et al. 1993. Identification and functional characterization of regulatory elements of the glutamine synthetase gene from rat liver. Eur J Biochem 213: 10671073.CrossRefGoogle ScholarPubMed
Fischer, A, Hacein-Bey-Abina, S, Cavazzana-Calvo, M. 2013. Gene therapy of primary T cell immunodeficiencies. Gene 525: 170173.CrossRefGoogle ScholarPubMed
Gouze, E, Gouze, JN, Palmer, GD, et al. 2007. Mol Ther 15: 11141120.CrossRefGoogle Scholar
Gumucio, JJ, May, M, Dvorak, C, Chianale, J, Massey, V. 1986. The isolation of functionally heterogeneous hepatocytes of the proximal and distal half of the liver acinus in the rat. Hepatology 6: 932944.CrossRefGoogle ScholarPubMed
Gumucio, JJ, Miller, DL. 1981. Functional implications of liver cell heterogeneity. Gastroenterology 80: 393403.CrossRefGoogle ScholarPubMed
Huh, YH, King, J, Cohen, J, Sherley, JL. 2011. SACK-expanded hair follicle stem cells display asymmetric nuclear lgr5 expression with non-random sister chromatid segregation. Sci Rep 1: 175.CrossRefGoogle ScholarPubMed
Jacomino, M, Lau, C, James, SZ, Henning, SJ. 1996. Gene transfer into fetal rat intestine. Hum Gene Ther 7: 17571762.CrossRefGoogle ScholarPubMed
Klein, AM, Simons, BD. 2011. Universal patterns of stem cell fate in cycling adult tissues. Development 138: 31033111.CrossRefGoogle ScholarPubMed
Le Lay, J, Kaestner, KH. 2010. The FOX genes in the liver: from organogenesis to functional integration. Physiol Rev 90: 122.CrossRefGoogle ScholarPubMed
Lee, H-S, Crane, GG, Merok, JR, et al. 2003. Clonal expansion of adult rat liver epithelial stem cells by suppression of asymmetric cell kinetics (SACK). Biotech Bioeng 83: 760771.CrossRefGoogle ScholarPubMed
Loeffler, M, Potten, CS. 1997. Stem cells and cellular pedigrees: a conceptual introduction. In Stem Cells, Potten, CS, ed., San Diego, CA: Harcourt Brace & Co., pp. 128.Google Scholar
MacDonald, RA. 1961. Lifespan of liver cells. Arch Intern Med 107: 335343.CrossRefGoogle ScholarPubMed
McClelland, R, Wauthier, E, Uronis, J, Reid, L. 2008a. Gradients in the liver’s extracellular matrix chemistry from periportal to pericentral zones: influence on human hepatic progenitors. Tissue Eng Part A 14: 5970.CrossRefGoogle ScholarPubMed
McClelland, R, Wauthier, E, Zhang, L, et al. 2008b. Ex vivo conditions for self-replication of human hepatic stem cells. Tissue Eng Part C 14: 341351.CrossRefGoogle ScholarPubMed
Messier, B, Leblond, CP. 1960. Cell proliferation and migration as revealed by radioautography after injection of thymidine-H3 into male rats and mice. Am J Anat 106: 247285.CrossRefGoogle ScholarPubMed
Miao, CH. 2016. Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors. Thrombosis J 14(Suppl. 1): 9399.CrossRefGoogle ScholarPubMed
Mukherjee, S, Thrasher, AJ. 2013. Gene therapy for PIDs: progress, pitfalls, and prospects. Gene 525: 174181.CrossRefGoogle ScholarPubMed
Nahon, J-L. 1987. The regulation of albumin and α-fetoprotein gene expression in mammals. Biochimie 69: 445459.CrossRefGoogle ScholarPubMed
Noh, M, Smith, JL, Huh, YH, Sherley, JL. 2011. A resource for discovering specific and universal biomarkers for distributed stem cells. PLoS One 6(7): e22077.CrossRefGoogle ScholarPubMed
Ouspenskaia, T, Matos, I, Mertz, AF, Fiore, VF, Fuchs, E. 2016. WNT-SHH antagonism specifies and expands stem cells prior to niche formation. Cell 164: 156169.CrossRefGoogle ScholarPubMed
Panchalingam, K, Noh, M, Hu, YH, Sherley, JL. 2016. Distributed stem cell kinetotoxicity: a new concept to account for the human carcinogenicity of non-genotoxic toxicants. In Human Stem Cell Toxicology, Issues in Toxicology No. 29, Sherley, JL, ed., London: Royal Society of Chemistry.Google Scholar
Paradinas, FJ, Bull, TB, Westaby, D, Murray-Lyon, IM. 1977. Hyperplasia and prolapse of hepatocytes into hepatic veins during longterm methyltestosterone therapy: possible relationships of these changes to the development of peliosis hepatis and liver tumors. Histopathology 1: 225246.CrossRefGoogle Scholar
Paré, J-F, Sherley, JL. 2013. Ex vivo expansion of human pancreatic distributed stem cells by suppression of asymmetric cell kinetics (SACK). J Stem Cell Res Ther 3: 149.Google Scholar
Potten, CS, Morris, RJ. 1988. Epithelial stem cells in vivo. J Cell Sci Suppl 10: 4562.CrossRefGoogle ScholarPubMed
Potten, CS, Schofield, R, Lajtha, LG. 1979. A comparison of cell replacement in bone marrow, testis, and three regions of surface epithelium. Biochim et Biophys Acta 560: 281299.Google ScholarPubMed
Rappaport, AM. 1973. The microcirculatory hepatic unit. Microvasc Res 6: 212228.CrossRefGoogle ScholarPubMed
Schmelzer, E, Wauthier, E, Reid, LM. 2006. The phenotypes of pluripotent human hepatic progenitors. Stem Cells 24: 18521858.CrossRefGoogle ScholarPubMed
Schmelzer, E, Zhang, L, Bruce, A, et al. 2007. Human hepatic stem cells from fetal and postnatal donors. J Exp Med 204: 19731987.CrossRefGoogle ScholarPubMed
Schwartz-Arad, D, Zajicek, G, Bartfeld, E. 1989. The streaming liver IV. DNA content of the hepatocyte increases with age. Liver 9: 9399.CrossRefGoogle Scholar
Sherley, JL. 2005. Asymmetric self-renewal: the mark of the adult stem cell. In Stem Cell Repair and Regeneration, Habib, NA, Gordon, MY, Levicar, N, Jiao, G, Thomas-Black, L, eds., London: Imperial College Press, pp. 2128.CrossRefGoogle Scholar
Sherley, JL. 2006. Mechanisms of genetic fidelity in mammalian adult stem cells. In Tissue Stem Cells, Potten, CS, Clarke, RB, Wilson, J, Renehan, AG, eds., New York: Taylor Francis, pp. 3754.Google Scholar
Sherley, JL. 2008. A new mechanism for aging: chemical “age spots” in immortal DNA strands in distributed stem cells. Breast Dis 29: 3746.CrossRefGoogle ScholarPubMed
Sherley, JL. 2013. New cancer diagnostics and therapeutics from a 9th “hallmark of cancer”: symmetric self-renewal by mutated distributed stem cells. Expert Rev Mol Diagn 13: 797810.CrossRefGoogle Scholar
Sherley, JL. 2016. Advancing stem cell medicine by supplying private stem cell clinics. Clinical Trials Arena Supply Chain. www.clinicaltrialsarena.com/news/supply-chain/advancing-stem-cell-medicine-by-supplying-private-stem-cell-clinics-5669514 (accessed November 2017).Google Scholar
Sherley, JL, Panchalingam, K. 2010. Methods for ex vivo propagation of adult hepatic stem cells. US Patent No. 7,824,912.Google Scholar
Sigal, SH, Brill, S, Fiorino, AS, Reid, LM. 1992. The liver as a stem cell and lineage system. Am J Physiol 263: G139G148.Google ScholarPubMed
Szybalski, W. 2013. The 50th anniversary of gene therapy: beginnings and present realities. Gene 525: 151154.CrossRefGoogle ScholarPubMed
Ungar, H. 1984. Primary portal venopathy in the golden hamster treated with low doses of dimethyl nitrosamine. Liver 4: 244254.CrossRefGoogle Scholar
Weber, GF. 2013. Gene therapy: why can it fail? Med Hypoth 80: 613616.CrossRefGoogle ScholarPubMed
Wirth, T, Parker, N, Ylä-Herttuala, S. 2013. History of gene therapy. Gene 525: 162169.CrossRefGoogle ScholarPubMed
Wright, N, Alison, M. 1984. The Biology of Epithelial Cell Populations, Vol. 2. Oxford: Clarendon Press, pp. 880980.Google Scholar
Xiong, X, Chen, M, Lim, WA, Zhao, D, Qi, LS. 2016. CRISPR/Cas9 for human genome engineering and disease research. Annu Rev Genom Hum Genet 17: 131154.CrossRefGoogle ScholarPubMed
Yu, K-R, Natanson, H, Dunbar, CE. 2016. Gene editing of human hematopoietic stem can progenitor cells: promise and potential hurdles. Hum Gene Ther 27: 729740.CrossRefGoogle ScholarPubMed
Zajicek, G, Arber, N, Schwartz-Arad, D. 1991. Streaming liver VIII: cell production rates following partial hepatectomy. Liver 11: 347351.CrossRefGoogle ScholarPubMed
Zajicek, G, Ariel, I, Arber, N. 1988. The streaming liver III. Littoral cells accompany the streaming hepatocyte. Liver 8: 213218.CrossRefGoogle ScholarPubMed
Zajicek, G, Oren, R, Weinreb, M Jr. 1985. The streaming liver. Liver 5: 293300.CrossRefGoogle ScholarPubMed
Zajicek, G, Swhwartz-Arad, D. 1990. Streaming liver VII: DNA turnover in acinus zone-3. Liver 10: 137140.CrossRefGoogle ScholarPubMed
Zajicek, G, Swhwartz-Arad, D, Bartfeld, E. 1989. The streaming liver V. Time and age-dependent changes of hepatocyte DNA content, following partial hepatectomy. Liver 9: 164171.CrossRefGoogle ScholarPubMed
Zepeda, ML, Chinov, MR, Wilson, JM. 1995. Characterization of stem cells in human airway capable of reconstituting a fully differentiated bronchial epithelium. Somat Cell Mol Genet 21: 6173.CrossRefGoogle ScholarPubMed
Zhou, S, Mody, D, DeRavin, SS, et al. 2010. A self-inactivating lentiviral vector for SCID-XI gene therapy that does not activate LMO2 expression in human T cells. Blood 116: 900908.CrossRefGoogle Scholar

Save book to Kindle

To save this book to your Kindle, first ensure coreplatform@cambridge.org is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about saving to your Kindle.

Note you can select to save to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be saved to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.

Find out more about the Kindle Personal Document Service.

Available formats
×

Save book to Dropbox

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Dropbox.

Available formats
×

Save book to Google Drive

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Google Drive.

Available formats
×