US 5756084A
· Honjo et al.
· 1998
[cited by applicant]
US 6448054B1
· Poznansky et al.
· 2002
[cited by applicant]
US 7033831B2
· Fisk et al.
· 2006
[cited by applicant]
US 7776564B2
· Chu et al.
· 2010
[cited by applicant]
US 8268620B2
· Thomson et al.
· 2012
[cited by applicant]
US 8546140B2
· Mack et al.
· 2013
[cited by applicant]
US 20040043040A1
· Dunussi-Joannopoulos
· 2004
[cited by applicant]
US 20070258943A1
· Penn et al.
· 2007
[cited by applicant]
US 20080213319A1
· Kang et al.
· 2008
[cited by applicant]
US 20090280096A1
· Kubo et al.
· 2009
[cited by applicant]
US 20120053119A1
· Habener et al.
· 2012
[cited by applicant]
US 20170196913A1
· Ben-Porath et al.
· 2017
[cited by applicant]
US 20190100729A1
· Kiewlich et al.
· 2019
[cited by applicant]
US 20190100730A1
· Swiss et al.
· 2019
[cited by applicant]
WO 2012025925A1
· 2012
[cited by applicant]
Transplantation, 2007, vol. 83, No. 2, pp. 174-183.
[cited by applicant]
United States Patent and Trademark Office, Notice of Allowance in U.S. Appl. No. 16/370,606 mailed Apr. 20, 2020.
[cited by applicant]
Urnov, F. D., et al., “Genome editing with engineered zinc finger nucleases,” Nature Reviews Genetics 11 (9): 636-646, 2010.
[cited by applicant]
Van Craenenbroeck, K., et al., “Episomal vectors for gene expression in mammalian cells,” European Journal of Biochemistry 267(18): 5665-5678, 2000.
[cited by applicant]
Vianello et al., “Fugetaxis: active movement of leukocytes away from a chemokinetic agent,” J. Mol. Med., 2005, 83:752-763.
[cited by applicant]
Votey, M., et al., “Of Mice and Men: How the NPOD Program Is Changing the Way Researchers Study Type 1 Diabetes,” DiaTribe, Aug. 21, 2015 (2 pages), diatribe.org/mice-and-men-how-npod-program-changing-wav-researchers-st…
[cited by applicant]
Walsh, N. C., et al., “Humanized mouse models of clinical disease,” Annual Review of Pathology: Mechanisms of Disease 12: 187-215, 2017.
[cited by applicant]
Wernig, M., et al., “In vitro reprogramming of fibroblasts into a pluripotent ES-cell-like state,” Nature 448(7151): 318-325, 2007.
[cited by applicant]
Xiao, H., et al., “Angelica sinensis Polysaccharides Ameliorate Stress-Induced Premature Senescence of Hematopoietic Cell via Protecting Bone Marrow Stromal Cells from Oxidative Injuries Caused by 5-Fluorouracil,” Int. …
[cited by applicant]
Yang, H., et al., “Human β Cells Are Exceedingly Resistant to Streptozotocin in Vivo,” Endocrinology, 2002, 143(7): 2491-2495.
[cited by applicant]
Yano, T., et al., “Stromal cell-derived factor-1 (SDF-1/CXCL 12) attenuates diabetes in mice and promotes pancreatic beta-cell survival by activation of the prosurvival kinase Akt,” Diabetes, 2007, 56(12): 2946-57.
[cited by applicant]
Yu, J., et al., “Induced pluripotent stem cell lines derived from human somatic cells,” Science 318: 1917-1920, 2007.
[cited by applicant]
Yu et al., “Identification and expression of novel isoforms of human stromal cell-derived factor 1,” Gene, 2006, 374: 174-179.
[cited by applicant]
Zhang, Z., et al., “CRISPR/Cas9 genome-editing system in human stem cells: current status and future prospects,” Molecular Therapy-Nucleic Acids 9: 230-241, 2017.
[cited by applicant]
Zhang, L., et al., “CXCL 12 overexpression promotes the angiogenesis potential of periodontal ligament stem cells,” Sci. Rep., 2017, 7: 10286.
[cited by applicant]
Zhong, Y., et al., “Targeting Interleukin-2-inducible T-cell Kinase (ITK) and Resting Lymphocyte Kinase (RLK) Using a Novel Covalent Inhibitor PRN694,” J. Biol. Chem., 2015, 290(10): 5960-5978.
[cited by applicant]
Mali, P., et al., “RNA-guided human genome engineering via Cas9,” Science 339(6121): 823-826, 2013.
[cited by applicant]
Massachusetts General Hospital, “Protein that repels immune cells protects transplanted pancreatic islets from rejection: Transplanting islets encapsulated with CXCL12 restores blood sugar control without immunosuppress…
[cited by applicant]
Mestas, J., et al., “Of mice and not men: differences between mouse and human immunology,” The Journal of Immunology, 2004, 172(5): 2731-2738.
[cited by applicant]
Miller, J.C., et al., “A TALE nuclease architecture for efficient genome editing,” Nature Biotechnology29(2): 143-150, 2010.
[cited by applicant]
Millette, K., et al., “Gene Editing and Human Pluripotent Stem Cells: Tools for Advancing Diabetes Disease Modeling and Beta-Cell Development,” Current Diabetes Reports 17(11): 116, 2017.
[cited by applicant]
Millman, J. R., et al., “Generation of stem cell-derived B-cells from patients with type 1 diabetes,” Nature Communications 7: 11463 (May 10, 2016) (9 pages).
[cited by applicant]
Naparstek, Y., et al., “Effector T lymphocyte line cells migrate to the thymus and persist there,” Nature, 1982, 300: 262-264. [Abstract only].
[cited by applicant]
Narayanavari, S. A., et al., “Sleeping Beauty transposon vectors for therapeutic applications: advances and challenges,” Cell and Gene Therapy Insights 3(2) (2017) (28 pages).
[cited by applicant]
Notice of Reasons for Rejection in related Japanese Application No. 2020-519249, pp. 1-13.
[cited by applicant]
Okita, K., et al., “Generation of germline-competent induced pluripotent stem cells,” Nature 448(7151): 313-318, 2007.
[cited by applicant]
Olivier, V., et al., “Comparative particle-induced cytotoxicity toward macrophages and fibroblasts,” Cell Biology and Toxicology, 2003, 19: 145-159.
[cited by applicant]
Orimo, A., et al., “Stromal Fibroblasts Present in Invasive Human Breast Carcinomas Promote Tumor Growth and Angiogenesis through Elevated SDF-1/CXCL 12 Secretion,” Cell, 2005, 121: 335-348.
[cited by applicant]
Orlando, G., et al., “Cell replacement strategies aimed at reconstitution of the β-cell compartment in type 1 diabetes,” Diabetes63(5): 1433-1444, 2014.
[cited by applicant]
Oumard, A., et al., “Recommended method for chromosome exploitation: RMCE-based cassette-exchange systems in animal cell biotechnology,” Cytotechnology 50.1-3 (2006): 93-108.
[cited by applicant]
Pagliuca, F. W., et al., “How to make a functional β-cell,” Development, 2013, 140(12): 2472-2483.
[cited by applicant]
Papeta, N., et al., “Long-term survival of transplanted allogeneic cells engineered to express a T cell chemorepellent,” Transplantation, 2007, 83(2): 174-183.
[cited by applicant]
Park, I., et al., “Reprogramming of human somatic cells to pluripotency with defined factors,” Nature 451: 141-145, 2008.
[cited by applicant]
Penn, M.S., et al., “SDF-1 in myocardial repair,” Gene Therapy, 2012, 19: 583-587.
[cited by applicant]
Pericin et al., “Allogeneic β-islet cells correct diabetes and resist immune rejection,” PNAS, 2002, 99(12): 8203-8206.
[cited by applicant]
Perl, S., et al., “Significant human β-cell turnover is limited to the first three decades of life as determined by in vivo thymidine analog incorporation and radiocarbon dating,” The Journal of Clinical Endocrinology &…
[cited by applicant]
Perlman, R., “Mouse models of human disease: An evolutionary perspective,” Eval. Med. Public Health, 2016, 2016 (1): 170-176.
[cited by applicant]
Petrova, N. V., et al., “Small molecule compounds that induce cellular senescence,” Aging Cell 15(6): 999-1017, 2016.
[cited by applicant]
PLOS ONE, 2013, vol. 8, Issue 3, e59679.
[cited by applicant]
Porteus, M. H., et al., “Gene targeting using zinc finger nucleases,” Nature Biotechnology 23(8): 967-973, 2005.
[cited by applicant]
Poznansky et al., “Thymocyte emigration is mediated by active movement away from stroma-derived factors,” J. Clin. Invest., 2002, 109(8): 1101-1110.
[cited by applicant]
Poznansky, M. C., et al., “Active movement of T cells away from a chemokine,” Nature Medicine 6(5): 543-548, 2000.
[cited by applicant]
Qadir, M. M. F., et al., “P2RY1/ALK3-Expressing Cells within the Adult Human Exocrine Pancreas Are BMP-7 Expandable and Exhibit Progenitor-like Characteristics,” Cell Reports 22(9): 2408-2420, 2018.
[cited by applicant]
Qi, M., “Transplantation of encapsulated pancreatic islets as a treatment for patients with type 1 diabetes mellitus,” Advances in Medicine, vol. 2014 (2014) (15 pages).
[cited by applicant]
Rabinovitch, A., et al., “Transfection of Human Pancreatic Islets With an Anti-Apoptotic Gene (bcl-2) Protects β-Cells From Cytokine-Induced Destruction,” Diabetes, 1999, 48(6): 1223-1229.
[cited by applicant]
Reply Brief filed Feb. 24, 2021 in U.S. Appl. No. 16/146,980.
[cited by applicant]
“Researchers find striking differences between human and animal insulin-producing islet cells,” Diabetes Research Institute Foundation Press Release, Feb. 2006, available at: https://www.diabetesresearch.org/differences…
[cited by applicant]
Reyon, D., et al., “FLASH assembly of TALENs for high-throughput genome editing,” Nature Biotechnology 30(5): 460-465, 2012.
[cited by applicant]
Ricordi, C., “Lilly Lecture 2002—Islet Transplantation: A Brave New World,” Diabetes, 2003, 52: 1595-1603.
[cited by applicant]
Roep, B. O., et al., “Animal models have little to teach us about Type 1 diabetes: 1. In support of this proposal,” Diabetologia, 2004, 47(10): 1650-1656.
[cited by applicant]
Route of administration. (n.d.). In Wikipedia. Retrieved Aug. 19, 2020, from https://en.wikipedia.ora/wiki/Route_of_administration.
[cited by applicant]
Russ, H. A., et al., “Controlled induction of human pancreatic progenitors produces functional beta-like cells in vitro,” The EMBO Journal 34(13): 1759-1772, 2015.
[cited by applicant]
Sakata, N., et al., “Encapsulated islets transplantation: past, present and future,” World Journal of Gastrointestinal Pathophysiology, 2012, 3(1): 19-26.
[cited by applicant]
Schuetz, C., et al., “Islet cell transplantation: update on current clinical trials,” Current Transplantation Reports 3(3): 254-263, 2016.
[cited by applicant]
Shi, Y., et al., “Inducing embryonic stem cells to differentiate into pancreatic β cells by a novel three-step approach with activin A and all-trans retinoic acid,” Stem Cells 23(5): 656-662, 2005.
[cited by applicant]
Sneddon et al., Stem Cell Therapies for Treating Diabetes; Progress and Remaining Challenges, Cell Stem Cell 2018, 22: 810-818.
[cited by applicant]
Sremac et al., “Preliminary Studies of the Impact of CXCL12 on the Foreign Body Reaction to Pancreatic Islets Microencapsulated in Alginate in Nonhuman Primates,” Transplantation Direct, 2019, 5(5): e447.
[cited by applicant]
Stewart, D., “CRISPR-Cas9 DNA Editing Possibly Linked to Cancer, But CRISPR-Cas13d RNA Editing Could Offer New Avenues for Treatment,” Dark Daily, Nov. 7, 2018, available at: https://www.darkdaily.com/crispr-cas9-dna-ed…
[cited by applicant]
Sundararaman, S., et al., “Plasmid-based transient human stromal cell-derived factor-1 gene transfer improves cardiac function in chronic heart failure,” Gene Therapy, 2011, 18: 867-873.
[cited by applicant]
Takahashi, K., et al., “Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors,” Cell 126(4): 663-676, 2006.
[cited by applicant]
Takahashi, K., et al., “Induction of pluripotent stem cells from adult human fibroblasts by defined factors,” Cell 131 (5): 861-872, 2007.
[cited by applicant]
Tateishi, K., et al., “Generation of insulin-secreting islet-like clusters from human skin fibroblasts,” Journal of Biological Chemistry 283(46): 31601-31607, 2008.
[cited by applicant]
Tatum, J. A., et al., “Single-donor islet transplantation in type 1 diabetes: patient selection and special considerations,” Diabetes, Metabolic Syndrome and Obesity: Targets and Therapy 10: 73-78, 2017.
[cited by applicant]
Teta, M., et al., “Very Slow Turnover of β-Cells in Aged Adult Mice,” Diabetes, 2005, 54: 2557-2567.
[cited by applicant]
The International Bureau of WIPO, International Preliminary Report on Patentability for International Application No. PCT/US2018/053599, mailed Apr. 16, 2020.
[cited by applicant]
Tipanee, J., et al., “Preclinical and clinical advances in transposon-based gene therapy,” Bioscience Reports 37(6) (2017) (20 pages).
[cited by applicant]
Aboumrad et al., “The CXCR4/CXCL 12 (SDF-1) signaling pathway protects non-obese diabetic mouse from autoimmune diabetes,” Clinical and Experimental Immunology, 2007, 148(3): 432-439.
[cited by applicant]
Altenburg, J., et al., “A Naturally Occurring Splice Variant of CXCL 12/Stromal Cell-Derived Factor 1 Is a Potent Human Immunodeficiency Virus Type 1 Inhibitor with Weak Chemotaxis and Cell Survival Activities,” J. Vira…
[cited by applicant]
Altieri, P., et al., “5-fluorouracil causes endothelial cell senescence: potential protective role of glucagon-like peptide 1,” British Journal of Pharmacology, 2017, 174: 3713-3726.
[cited by applicant]
Ansari, A., et al., “Cellular GFP Toxicity and Immunogenicity: Potential Confounders in in Vivo Cell Tracking Experiments,” Stem Cell Rev., 2016, 12(5): 553-559.
[cited by applicant]
Appeal Brief filed Nov. 20, 2020 in U.S. Appl. No. 16/146,980.
[cited by applicant]
Baek, K., et al., “Gene transfection for stem cell therapy,” Current Stem Cell Reports 2(1): 52-61, 2016.
[cited by applicant]
Berezhnoi, A. E., et al., “High-dose Gamma-Irradiation Enhance Expression of Transgene Under Control of Immediate-Early CMV Promoter in Stably Transfected Tumor Cells,” Mol. Biol. (Mosk), 2008, 42(3): 501-509. [Abstract…
[cited by applicant]
Bhatt, J.M., et al., “Expression of Epitope-Tagged Proteins in Mammalian Cells in Culture,” Methods Mol. Biol., 2016, 1474: 3-24 [Abstract].
[cited by applicant]
Brehm, M. A., et al., “Advancing animal models of human type 1 diabetes by engraftment of functional human tissues in immunodeficient mice,” Cold Spring Harbor Perspectives in Medicine 2(5): a007757, 2012.
[cited by applicant]
Cabrera, O ., et al., “The unique cytoarchitecture of human pancreatic islets has implications for islet cell function,” Proceedings of the National Academy of Sciences 103(7): 2334-2339, 2006.
[cited by applicant]
Cerignoli, F., et al., “In vitro immunotherapy potency assays using real-time cell analysis,” PLOS One, 2018, available at: https://doi.org/10.1371/journal.pone.0193498.
[cited by applicant]
Cermak, T., et al., “Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting,” Nucleic Acids Research 39(12): e82, 2011 (11 pages).
[cited by applicant]
Charles, M.A., et al., “Immune islet killing mechanisms associated with insulin-dependent diabetes: in vitro expression of cellular and antibody-mediated islet cell cytotoxicity in humans,” J. Immunol., 1983, 130(3): 11…
[cited by applicant]
Chira, S., et al., “Progresses towards safe and efficient gene therapy vectors,” Oncotarget 6(31): 30675-30703, 2015.
[cited by applicant]
Chong et al., “Lessons and Limits of Mouse Models,” Cold Spring Harb. Perspect. Med., 2013, 3(12): 1-16.
[cited by applicant]
“Cloning Technique Used to Create Pancreatic Cells for Type 1 Diabetes.” Diabetes Community, Support, Education, Recipes & Resources, Apr. 29, 2014 (4 pages), www.diabetes.co.uknews/2014/apr/cloning-technique-used-to-cr…
[cited by applicant]
Collier, J. J., et al., “Pro-and antiapoptotic proteins regulate apoptosis but do not protect against cytokine-mediated cytotoxicity in rat islets and β-cell lines,” Diabetes 55(5): 1398-1406, 2006.
[cited by applicant]
Cong, L., et al., “Multiplex genome engineering using CRISPR/Cas systems,” Science (2013): 819-823.
[cited by applicant]
Copenhaver, M., et al., “Type 1 diabetes: where are we in 2017?,” Transl. Pediatr., 2017, 6(4): 359-364.
[cited by applicant]
El-Haibi, C.P., et al., “CXCL 13 mediates prostate cancer cell proliferation through JNK signalling and invasion through ERK activation,” Cell Proliferation 44(4) (2011) (16 pages).
[cited by applicant]
European Patent Office, International Search Report and Written Opinion for International Application No. PCT/US2018/053599, mailed Nov. 28, 2018.
[cited by applicant]
Examiner's Answer mailed Dec. 28, 2020 in in U.S. Appl. No. 16/146,980.
[cited by applicant]
Final Office Action mailed Jun. 29, 2020 in U.S. Appl. No. 16/146,980.
[cited by applicant]
Final Office Action dated Jan. 31, 2023 for U.S. Appl. No. 16/633,744, 9 pgs.
[cited by applicant]
Focaccetti, C., et al., “Effects of 5-Fluorouracil on Morphology, Cell Cycle, Proliferation, Apoptosis, Autophagy and ROS Production in Endothelial Cells and Cardiomyocytes,” PLoS ONE, 2015, 10(2): e0115686. doi: 10.137…
[cited by applicant]
GenBank E09668.1, cDNA encoding human SDF-1 alpha, 2005 (2 pages).
[cited by applicant]
GenBank E09669.1, cDNA of human SDF-1 beta isoform, 2005 (2 pages).
[cited by applicant]
Gonzalez, F., et al., “An iCRISPR platform for rapid, multiplexable, and inducible genome editing in human pluripotent stem cells,” Cell Stem Cell 15(2): 215-226, 2014.
[cited by applicant]
Goping, I. S., et al., “Cytotoxic T lymphocytes overcome Bcl-2 inhibition: target cells contribute to their own demise,” Blood 111 (4): 2142-2151, 2008.
[cited by applicant]
Götherström, C., et al., “Fetal and adult multipotent mesenchymal stromal cells are killed by different pathways,” Cytotherapy, 2011, 13(3): 269-278.
[cited by applicant]
Hardee, C.L., et al. “Advances in non-viral DNA vectors for gene therapy,” Genes 8(65) (2017) (22 pages).
[cited by applicant]
He, X., et al., “Knock-in of large reporter genes in human cells via CRISPR/Cas9-induced homology-dependent and independent DNA repair,” Nucleic Acids Research 44(9) (2016): e85-e85.
[cited by applicant]
Helman, A., et al., “p161nk4a_induced senescence of pancreatic beta cells enhances insulin secretion,” Nature Medicine, 2016, 22(4): 412-422.
[cited by applicant]
Herberts, C., et al., “Risk factors in the development of stem cell therapy,” J. Transl. Med., 2011, 9: 29.
[cited by applicant]
Hockemeyer, D., et al., “Genetic engineering of human pluripotent cells using TALE nucleases,” Nature Biotechnology 29(8): 731, 2011.
[cited by applicant]
Hsu, P. D., et al., “Development and applications of CRISPR-Cas9 for genome engineering,” Cell 157(6): 1262-1278, 2014.
[cited by applicant]
Ilhan, A., et al., “CXCL12/SDF-1 over-expression in human insulinomas and its biological relevance,” Molecular and Cellular Endocrinology, 2009, 298: 1-10.
[cited by applicant]
Johannesson, B., et al., “Toward beta cell replacement for diabetes,” The EMBO Journal 34(7): 841-855, 2015.
[cited by applicant]
Joung, J. K., et al., “TALENs: a widely applicable technology for targeted genome editing,” Nature Reviews Molecular Cell Biology 14(1): 49-55, 2013.
[cited by applicant]
Kaja, S., et al., “Quantification of Lactate Dehydrogenase for Cell Viability Testing Using Cell Lines and Primary Cultured Astrocytes,” Current Protocols in Toxicology, 2017, Suppl 72: 2.26.1-2.26.10.
[cited by applicant]
Kelly, O. G., et al., “Cell-surface markers for the isolation of pancreatic cell types derived from human embryonic stem cells,” Nature Biotechnology 29(8) (2011) (9 pages).
[cited by applicant]
Kiewlich Declaration signed Feb. 7, 2020, and submitted in U.S. Appl. No. 16/146,980.
[cited by applicant]
Kiewlich Declaration signed Nov. 20, 2019, and submitted in U.S. Appl. No. 16/370,606.
[cited by applicant]
King, A., “The use of animal models in diabetes research,” British Journal of Pharmacology, 2012, 166: 877-894.
[cited by applicant]
Kroon, E., et al., “Pancreatic endoderm derived from human embryonic stem cells generates glucose-responsive insulin-secreting cells in vivo,” Nature Biotechnology 26(4): 443-452, 2008.
[cited by applicant]
Leach, C., “The Mouse Trap,” Insulin Nation, Nov. 18, 2013, available at: https://insulinnation.com/treatment/cure-insight/the-mouse-trap/.
[cited by applicant]
Liang, X., et al., “Human bone marrow mesenchymal stem cells expressing SDF-1 promote hematopoietic stem cell function of human mobilised peripheral blood CD34+ cells in vivo and in vitro,” Int. J. Radiat. Biol., 2010, …
[cited by applicant]
Lowry, W. E., et al., “Generation of human induced pluripotent stem cells from dermal fibroblasts,” Proceedings of the National Academy of Sciences 105(8): 2883-2888, 2008.
[cited by applicant]
Luther, S. A., et al., “Differing activities of homeostatic chemokines CCL 19, CCL21, and CXCL 12 in lymphocyte and dendritic cell recruitment and lymphoid neogenesis,” The Journal of Immunology 169(1): 424-433, 2002.
[cited by applicant]
Maherali, N., et al., “Directly reprogrammed fibroblasts show global epigenetic remodeling and widespread tissue contribution,” Cell Stem Cell 1(1): 55-70, 2007.
[cited by applicant]