Granted Patent
B2
US 12,227,768 · App. 17/958,816 · Granted Feb 18, 2025
Methods and products for transfection
Assignee:
Factor Bioscience Inc.
C12N5/0696C12N5/0625C12N5/0647C12N5/0656C12N5/0657C12N5/0676C12N5/0695C12N15/85C12N5/0018C12N2500/25C12N2500/33C12N2500/36C12N2500/84C12N2501/602C12N2501/603C12N2501/604C12N2501/606C12N2501/608C12N2501/998C12N2506/09C12N2506/45
View Patent ↗
Loading inventors, assignments & file history…
Abstract
The present invention relates in part to methods for producing tissue-specific cells from patient samples, and to tissue-specific cells produced using these methods. Methods for reprogramming cells using RNA are disclosed. Therapeutics comprising cells produced using these methods are also disclosed.
Claims (32)
1. A method for reprogramming a differentiated cell to a less differentiated state, the method comprising:
(a) providing the differentiated cell;
(b) culturing the differentiated cell; and
(c) transfecting the differentiated cell with one or more synthetic RNA molecules,
wherein the one or more synthetic RNA molecules include at least one RNA molecule encoding one or more reprogramming factors comprising a DNA-binding protein; and
wherein the transfecting results in the cell expressing the one or more reprogramming factors to result in the cell being reprogrammed to the less differentiated state; and
wherein step (c) is performed not in the presence of irradiated fibroblast feeder cells and occurs in the presence of a medium containing ingredients that support reprogramming of the differentiated cell to the less differentiated state.
2. The method of claim 1 , wherein the differentiated cell is derived from a biopsy.
3. The method of claim 1 , wherein the differentiated cell is derived from a dermal punch biopsy sample.
4. The method of claim 1 , wherein the differentiated cell is from a human subject.
5. The method of claim 1 , wherein the differentiated cell is a skin cell.
6. The method of claim 1 , further comprising contacting the differentiated cell with at least one member selected from the group consisting of: poly-L-lysine, poly-L-ornithine, RGD peptide, fibronectin, vitronectin, collagen, and laminin.
7. The method of claim 1 , wherein the at least one synthetic RNA molecule contains at least one of a pseudouridine residue or a 5-methylcytidine residue.
8. The method of claim 1 , wherein steps (b) and (c) occur in the presence of the medium containing ingredients that support reprogramming of the differentiated cell.
9. The method of claim 1 , wherein steps (b) and (c) are performed not in the presence of irradiated human neonatal fibroblast feeder cells.
10. The method of claim 8 , wherein the medium in the presence of which steps (b) and (c) occur is substantially free of immunosuppressants.
11. A method for reprogramming a non-pluripotent cell, the method comprising:
(a) providing the non-pluripotent cell;
(b) culturing the non-pluripotent cell; and
(c) transfecting the non-pluripotent cell with one or more synthetic RNA molecules,
wherein the one or more synthetic RNA molecules include at least one RNA molecule encoding one or more reprogramming factors comprising a DNA-binding protein; and
wherein the transfecting results in the cell expressing the one or more reprogramming factors to result in the cell being reprogrammed; and
wherein step (c) is performed not in the presence of irradiated fibroblast feeder cells and occurs in the presence of a medium containing ingredients that support reprogramming of the non-pluripotent cell.
12. The method of claim 11 , wherein the non-pluripotent cell is derived from a biopsy.
13. The method of claim 11 , wherein the non-pluripotent cell is derived from a dermal punch biopsy sample.
14. The method of claim 11 , wherein the non-pluripotent cell is from a human subject.
15. The method of claim 11 , wherein the non-pluripotent cell is a skin cell.
16. The method of claim 11 , further comprising contacting the non-pluripotent cell with at least one member selected from the group consisting of: poly-L-lysine, poly-L-ornithine, RGD peptide, fibronectin, vitronectin, collagen, and laminin.
17. The method of claim 11 , wherein the at least one synthetic RNA molecule contains at least one of a pseudouridine residue or a 5-methylcytidine residue.
18. The method of claim 11 , wherein steps (b) and (c) occur in the presence of the medium containing ingredients that support reprogramming of the non-pluripotent cell.
19. The method of claim 11 , wherein steps (b) and (c) are performed not in the presence of irradiated human neonatal fibroblast feeder cells.
20. The method of claim 18 , wherein the medium in the presence of which steps (b) and (c) occur is substantially free of immunosuppressants.
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Feb 2, 2023
From: ANGEL, MATTHEW; ROHDE, CHRISTOPHER
To: FACTOR BIOSCIENCE INC.
Reel/Frame 062571/0758 →
Continuity (15)
Provisional Application
61637570
· Apr 24, 2012
Provisional Application
61569595
· Dec 12, 2011
Provisional Application
61566948
· Dec 5, 2011
References Cited (316)
US 2705230A
· Reid
· 1955
[cited by applicant]
US 3539465A
· Hiestand et al.
· 1970
[cited by applicant]
US 5843780A
· Thomson
· 1998
[cited by applicant]
US 7276489B2
· Agrawal et al.
· 2007
[cited by applicant]
US 7442548B2
· Thomson et al.
· 2008
[cited by applicant]
US 7449334B2
· Thomson et al.
· 2008
[cited by applicant]
US 7621606B2
· Page et al.
· 2009
[cited by applicant]
US 7682828B2
· Jaenisch et al.
· 2010
[cited by applicant]
US 7687266B2
· Chambers et al.
· 2010
[cited by applicant]
US 7812000B2
· Agrawal et al.
· 2010
[cited by applicant]
US 8048675B1
· Irion
· 2011
[cited by applicant]
US 8048999B2
· Yamanaka et al.
· 2011
[cited by applicant]
US 8058065B2
· Yamanaka et al.
· 2011
[cited by applicant]
US 8071369B2
· Jaenisch et al.
· 2011
[cited by applicant]
US 8129187B2
· Yamanaka et al.
· 2012
[cited by applicant]
US 8202850B2
· Agrawal et al.
· 2012
[cited by applicant]
US 8278036B2
· Kariko et al.
· 2012
[cited by applicant]
US 8420782B2
· Bonas et al.
· 2013
[cited by applicant]
US 8440431B2
· Voytas et al.
· 2013
[cited by applicant]
US 8440432B2
· Voytas et al.
· 2013
[cited by applicant]
US 8450471B2
· Voytas et al.
· 2013
[cited by applicant]
US 8470973B2
· Bonas et al.
· 2013
[cited by applicant]
US 8497124B2
· Angel et al.
· 2013
[cited by applicant]
US 8586526B2
· Gregory et al.
· 2013
[cited by applicant]
US 8691966B2
· Kariko et al.
· 2014
[cited by applicant]
US 8709492B2
· Teschner et al.
· 2014
[cited by applicant]
US 8710200B2
· Schrum et al.
· 2014
[cited by applicant]
US 8716465B2
· Rossi et al.
· 2014
[cited by applicant]
US 8748089B2
· Kariko et al.
· 2014
[cited by applicant]
US 8802438B2
· Rossi et al.
· 2014
[cited by applicant]
US 8822663B2
· Schrum et al.
· 2014
[cited by applicant]
US 8835108B2
· Kariko et al.
· 2014
[cited by applicant]
US 8883506B2
· Rossi et al.
· 2014
[cited by applicant]
US 9399761B2
· Angel et al.
· 2016
[cited by applicant]
US 9422577B2
· Angel et al.
· 2016
[cited by applicant]
US 9562218B2
· Angel et al.
· 2017
[cited by applicant]
US 9605278B2
· Angel et al.
· 2017
[cited by applicant]
US 9695401B2
· Angel et al.
· 2017
[cited by applicant]
US 9879228B2
· Angel et al.
· 2018
[cited by applicant]
US 9969983B2
· Angel et al.
· 2018
[cited by applicant]
US 20030083272A1
· Wiederholt et al.
· 2003
[cited by applicant]
US 20050053588A1
· Yin
· 2005
[cited by applicant]
US 20050130144A1
· Nakatsuji et al.
· 2005
[cited by applicant]
US 20070134796A1
· Holmes et al.
· 2007
[cited by applicant]
US 20090029465A1
· Thomson et al.
· 2009
[cited by applicant]
US 20090093433A1
· Woolf et al.
· 2009
[cited by applicant]
US 20090180996A1
· Beyhan et al.
· 2009
[cited by applicant]
US 20090275128A1
· Thomson et al.
· 2009
[cited by applicant]
US 20100047261A1
· Hoerr et al.
· 2010
[cited by applicant]
US 20100075421A1
· Yamanaka et al.
· 2010
[cited by applicant]
US 20100076057A1
· Sontheimer et al.
· 2010
[cited by applicant]
US 20100093090A1
· Deng et al.
· 2010
[cited by applicant]
US 20100120079A1
· Page et al.
· 2010
[cited by applicant]
US 20100168000A1
· Kiessling et al.
· 2010
[cited by applicant]
US 20100172882A1
· Glazer et al.
· 2010
[cited by applicant]
US 20100184033A1
· West et al.
· 2010
[cited by applicant]
US 20100272695A1
· Agulnick et al.
· 2010
[cited by applicant]
US 20100273220A1
· Yanik et al.
· 2010
[cited by applicant]
US 20110045001A1
· Klosel et al.
· 2011
[cited by applicant]
US 20110065103A1
· Sahin et al.
· 2011
[cited by applicant]
US 20110171185A1
· Klimanskaya et al.
· 2011
[cited by applicant]
US 20110189137A1
· Rana
· 2011
[cited by applicant]
US 20110236978A1
· Stolzing et al.
· 2011
[cited by applicant]
US 20110239315A1
· Bonas et al.
· 2011
[cited by applicant]
US 20110263015A1
· D'Costa et al.
· 2011
[cited by applicant]
US 20120195936A1
· Rudolph et al.
· 2012
[cited by applicant]
US 20120237975A1
· Schrum et al.
· 2012
[cited by applicant]
US 20130040302A1
· Burke et al.
· 2013
[cited by applicant]
US 20130071365A1
· Suzuki
· 2013
[cited by applicant]
US 20130102034A1
· Schrum
· 2013
[cited by applicant]
US 20130123481A1
· De Fougerolles et al.
· 2013
[cited by applicant]
US 20130156849A1
· De Fougerolles et al.
· 2013
[cited by applicant]
US 20130165504A1
· Bancel et al.
· 2013
[cited by applicant]
US 20130189741A1
· Meis et al.
· 2013
[cited by applicant]
US 20130217119A1
· Bonas et al.
· 2013
[cited by applicant]
US 20130244282A1
· Schrum et al.
· 2013
[cited by applicant]
US 20130245103A1
· De Fougerolles et al.
· 2013
[cited by applicant]
US 20140073053A1
· Yanik et al.
· 2014
[cited by applicant]
US 20140127814A1
· Chandrasegaran et al.
· 2014
[cited by applicant]
US 20140242154A1
· Ramunas et al.
· 2014
[cited by applicant]
US 20140242595A1
· Yu et al.
· 2014
[cited by applicant]
CN 101432031A
· 2009
[cited by applicant]
EP 2241572A2
· 2010
[cited by applicant]
FR 2322871A1
· 1977
[cited by applicant]
JP 2001500867A
· 2001
[cited by applicant]
JP 2005536452A
· 2005
[cited by applicant]
JP 2009502124A
· 2009
[cited by applicant]
JP 2010529851A
· 2010
[cited by applicant]
JP 2010246551A
· 2010
[cited by applicant]
JP 2011160661A
· 2011
[cited by applicant]
WO WO9812225A2
· 1998
[cited by applicant]
WO WO9830679A1
· 1998
[cited by applicant]
WO WO0044772A2
· 2000
[cited by applicant]
WO WO0226757A2
· 2002
[cited by applicant]
WO WO03066681A1
· 2003
[cited by applicant]
WO WO2007012009A1
· 2007
[cited by applicant]
WO WO2007024708A2
· 2007
[cited by applicant]
WO WO2008065381A1
· 2008
[cited by applicant]
WO WO2009007852A2
· 2009
[cited by applicant]
WO WO2009077134A2
· 2009
[cited by applicant]
WO WO2009123349A1
· 2009
[cited by applicant]
WO WO2009127230A1
· 2009
[cited by applicant]
WO WO2009147400A1
· 2009
[cited by applicant]
WO WO2010093655A2
· 2010
[cited by applicant]
WO WO2010123501A1
· 2010
[cited by applicant]
WO WO2011011767A1
· 2011
[cited by applicant]
WO WO2011012316A2
· 2011
[cited by applicant]
WO WO2011058558A2
· 2011
[cited by applicant]
WO WO2011071931A2
· 2011
[cited by applicant]
WO WO2011071936A2
· 2011
[cited by applicant]
WO WO2011072246A2
· 2011
[cited by applicant]
WO WO2011094738A1
· 2011
[cited by examiner]
WO WO2011110886A1
· 2011
[cited by applicant]
WO WO2011114237A2
· 2011
[cited by applicant]
WO WO2011130624A2
· 2011
[cited by applicant]
WO WO2011132799A1
· 2011
[cited by applicant]
WO WO2011134210A1
· 2011
[cited by applicant]
WO WO2011139336A1
· 2011
[cited by applicant]
WO WO2011140397A2
· 2011
[cited by applicant]
WO WO2011141820A1
· 2011
[cited by applicant]
WO WO2011146121A1
· 2011
[cited by applicant]
WO WO2011154393A1
· 2011
[cited by applicant]
WO WO2012019122A2
· 2012
[cited by applicant]
WO WO2012019168A2
· 2012
[cited by applicant]
WO WO2012036299A1
· 2012
[cited by applicant]
WO WO2012048213A1
· 2012
[cited by applicant]
WO WO2012060473A1
· 2012
[cited by applicant]
WO WO2012122318A2
· 2012
[cited by applicant]
WO WO2012138453A1
· 2012
[cited by applicant]
WO WO2013003475A1
· 2013
[cited by applicant]
WO WO2013102203A1
· 2013
[cited by applicant]
WO WO2013151671A1
· 2013
[cited by applicant]
WO WO2013163296A1
· 2013
[cited by applicant]
WO WO2013173248A2
· 2013
[cited by applicant]
WO WO2014190361A2
· 2014
[cited by applicant]
Han and Yoon (Antioxidants & Redox Signaling 15(7):1799-1820) (Year: 2011).
[cited by examiner]
Anderson, B.R.: Nucleoside modifications suppress RNA activation of cytoplasmic RNA sensors. Publicly Accessible Penn Dissertations. (Fall 2010).
[cited by applicant]
Anderson et al_, “Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation,” Nucl. Acids Res. 38(17): 1-9 (2010).
[cited by applicant]
Anderson et al, “Nucleofection induces transient eiF2a phosphorylation by GCN2 and PERK,” Gene Ther. 1-7 (2012).
[cited by applicant]
Anderson et al_, “Nucleoside modifications in RNA limit activation of 2′-5′-oligoadenylate synthetase and increase resistance to cleavage by RNase L,” Nucl. Acids Res. 39(21): 9329-9338 (2011).
[cited by applicant]
Angel et at, “Innate Immune Suppression Enables Frequent Transfection with RNA Encoding Reprogramming Proteins,” PLoS One 5(7): e11756, pp. 1-7 (Jul. 2010).
[cited by applicant]
Angel, “Extended Transient Transfection by Repeated Delivery of an In Vitro-Transcribed RNA,” Master of Science in Electrical Engineering and Computer Science, 56 pages (Massachusetts Institute of Technology, Cambridge,…
[cited by applicant]
Angel, “Reprogramming human somatic cells to pluripotency using RNA,” Doctor of Philosophy in Electrical Engineering and Computer Science, 55 pages (Massachusetts Institute of Technology, Cambridge, Massachusetts) (Oct.…
[cited by applicant]
Angel, “Reprogramming Human Somatic Cells to Pluripotency Using RNA”, pp. 1-89 (Phil diss., Massachusetts Institute of Technology) (Feb. 2012.
[cited by applicant]
Arnold et al., “Reprogramming of Human Huntington Fibroblasts Using mRNA,” ISRN Cell Biology Article ID 124878: 1-12 (2012).
[cited by applicant]
Barker et al., “A method for the deionization of bovine serum albumin,” Tissue Culture Association pp. 111-112 (1975).
[cited by applicant]
Berg, “Proposed structure for the zinc-binding domains from transcription factor IIIA and related proteins,” Proc. Natl. Acad. Sci. USA, 85: 99-102 (1988).
[cited by applicant]
Boch et al., “Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors,” Science 3126: 1509-1512 (2009).
[cited by applicant]
Bogdanove AJ, et al., “TAL Effectors: Customizable Proteins for DNA Targeting”, Science, 2011, vol. 333, No. 6051, pp. 1843-1846.
[cited by applicant]
Bolli et al., “Cardiac stem cells in patients with ischaemic cardiomyopathy (SCIPIO): initial results of a randomised phase 1 trial,” Lancet 1-11 (2011).
[cited by applicant]
Braam et al., “Recombinant vitronectin is a functionally defined substrate that supports human embryonic stem cell self-renewal via au 135 integrin,” Stem Cells 26: 2257-2265 (2008).
[cited by applicant]
Brazilian Patent Application No. 12 2021 023330-6 Office Action dated Jun. 27, 2023.
[cited by applicant]
Carroll, Progress and prospects: Zinc-finger nucleases as gene therapy agents, Gene Therapy 15: 1463-1468 (2008).
[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, 2011, vol. 39, No. 12, e82, pp. 1-11.
[cited by applicant]
Chen et al., “Rational optimization of reprogramming culture conditions for the generation of induced pluripotent stem mils with ultra-high efficiency and fast kinetics,” Cell Research 21: 884-894 (2011).
[cited by applicant]
Chen et al., “Role of MEF Feeder Cells in Direct Reprogramming of Mouse tail-tip Fibroblasts”, Cell Biology, 2009, vol. 33, No. 12, pp. 1268-1273.
[cited by applicant]
Chen, Guokai et al. Chemically Defined Conditions for Human iPSC Derivation and Culture. Nature Methods vol. 8,5: pp. 424-431 (2011).
[cited by applicant]
Christian et al., Targeting DNA Double-Strand Breaks with TAL Effector Nucleases, Genetics 186: 757-761 (2010).
[cited by applicant]
Christian, et al. Targeting DNA double-strand breaks with TAL effector nucleases. Genetics.186 (2010): 757-761.
[cited by applicant]
Cui et al., “Targeted integration in rat and mouse embryos with zinc-finger nucleases,” Nat. Biotech. 29(1):54-67 (2011).
[cited by applicant]
Davis, “Stabilization of RNA stacking by pseudouridine,” Nucleic Acids Research 23(24): 5020-5026 (1995).
[cited by applicant]
Droge et al., “A comparative study of some physico-chemical properties of human serum albumin samples from different sources—I. Some physico-chemical properties of isotonic human serum albumin solutions,” Biochem. Pharm…
[cited by applicant]
Efe et al., “Conversion of mouse fibroblasts into cardiomyocytes using a direct reprogramming strategy,” Nat. Cell Biol 13: 215-222 (2011).
[cited by applicant]
EP Application No. 20211670 Search Report dated May 17, 2021.
[cited by applicant]
Fusaki et al., Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome. Proc Jpn Acad Ser B Phys Biol Sci 85(8):…
[cited by applicant]
Garcia-Gonzalo et al., “Albumin-associated lipids regulate human embryonic stem cell self-renewal,” PLoS One 3: e1384, pp. 1-10 (2008).
[cited by applicant]
Geurts et al., “Knockout Rats via Embryo Microinjection of Zinc-Finger Nucleases,” Science 325: 433 (2009).
[cited by applicant]
Gibco Albumax I product insert, Invitrogen Corporation (Jun. 2001).
[cited by applicant]
Goldberg et al., “The enzymic synthesis of pseudouridine triphosphate,” Biochim. Biophys. Acta 54: 202-204(1961).
[cited by applicant]
Goldberg et al., “The incorporation of 5-ribosyluracil triphosphate into RNA in nuclear extracts of mammalian cells,” Biochim. Biophys. Res. Commun 6: 394-398 (1961).
[cited by applicant]
Goldberg, “Ribonucleic acid synthesis in nuclear extracts of mammalian cells grown in suspension culture; effect of Ionic strength and surface-active agents,” Biochim. Biophys. Acta 51: 201-204 (1961).
[cited by applicant]
Greenberg J.R., “High Stability of Messenger RNA in Growing Cultured Cells”, Nature, Nov. 10, 1972, vol. 240:102-104.
[cited by applicant]
Gurung et al., Beta-Catenin Is a Mediator of the Response of Fibroblasts to Irradiation, The American Journal of Pathology 174(1): 248-255 (2009).
[cited by applicant]
Hamanaka et al., “Generation of Germline-Component Rat Induced Pluripotent Stem Cells,” PlosOne 6(7): 1-9 (2011).
[cited by applicant]
Han, Ji, et al., Induced Pluripotent Stem Cells: Emerging Techniques for Nuclear Reprogramming. Antioxid Redox Signal 15(7):1799-820(2011).
[cited by applicant]
Hockemeyer et al., “Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases,” Nature Biotechnology 27(9): 851-857 (2009).
[cited by applicant]
Hockemeyer et al., “Genetic engineering of human ES and iPS cells using TALE nucleases,” Author Manuscript, available in PMC Feb. 1, 2012. Published in final edited form as: Nat Biotechnol. 29(8): 731-734 (2012).
[cited by applicant]
Hockmeyer, D, et al., “Genetic engineering of human pluripotent cells using TALE nucleases”, Nat Biotechnol, Jul. 7, 2011, vol. 29, No. 8, pp. 731-734.
[cited by applicant]
Holt et al., Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nature Biotechnology 28(8): 839-847 (2010).
[cited by applicant]
Holt, N., et al., “Zinc finger nuclease-mediated CCR5 knockout hematopoietic stem cell transplantation controls HIV-1 in vivo”, Nat Biotechnol., Aug. 2010; 28(8):839-47, doi:10.1038/nbt.1663.
[cited by applicant]
Huang et al.: Heritable gene targeting in zebrafish using customized TALENs. Nature Biotechnology. 29(8):699-700 (2011).
[cited by applicant]
Jasin et al.: Democratization of gene editing: Insights from site-specific cleavage and double-strand break repair. DNA Report (Amst) 44:6-16 (Aug. 2016).
[cited by applicant]
Kahan et al., “The Role of Deoxyribonucleic Acid in Ribonucleic Acid Synthesis,” The Journal of Biological Chemistry 237(12): 3778-3785 (1962).
[cited by applicant]
Kariko et al. Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA. Nucleic Acids Res. 39:e142-e142 (2011).
[cited by applicant]
Kariko et al., “In vivo protein expression from mRNA delivered into adult rat brain,” J. Neurosci. Methods 105: 17-86 (2001).
[cited by applicant]
Kariko et al., “Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability,” Mol. Ther 16: 1833-1840 (2008).
[cited by applicant]
Kariko et al., “Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin,” Mol. Ther. 20: 948-953 (2012).
[cited by applicant]
Kariko et al., “mRNA is an endogenous ligand for Toll-like receptor 3,” J. Biol. Chem. 279: 12542-12550 (2004).
[cited by applicant]
Kariko et al., Naturally occurring nucleoside modifications suppress the immunostimulatory activity of RNA: Implication for therapeutic RNA development. Drug Discovery & Development 10(5): 523-532 (2007).
[cited by applicant]
Kariko et al., Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA. Immunity 23: 165-175 (2005).
[cited by applicant]
Kawamata et al., Generation of genetically modified rats from embryonic stem cells. PNAS 107(32): 14223-14228 (2010).
[cited by applicant]
Kim et al., Direct reprogramming of human neural stem cells by OCT4. Nature 461: 649-653 (2009).
[cited by applicant]
Kim et al., Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins. Cell Stem Cell 4: 472-476 (2009).
[cited by applicant]
Kim et al., Hybrid restriction enzymes: Zinc finger fusions to Fok I cleavage domain. PNAS USA 93:1156-1160 (1996).
[cited by applicant]
Kim et al., Oct4-induced pluripotency in adult neural stem cells. Cell 136: 411-419 (2009).
[cited by applicant]
Kim et al., Pluripotent stem cells induced from adult neural stem cells by reprogramming with two factors. Nature 454: 1-6 (2008).
[cited by applicant]
Kormann, D. et al., “Expression of therapeutic proteins after delivery of chemically modified mRNA in mice”, Nature Biotechnology, 2011, vol. 29, No. 2, pp. 154-157.
[cited by applicant]
Lee et al., “Activation of Innate Immunity Is Required for Efficient Nuclear Reprogramming,” Cell 151: 547-558 (2012).
[cited by applicant]
Li et al.: An apolipoprotein E-mimetic stimulates axonal regeneration and remyelination after peripheral nerve injury. J. Pharm. & Experimental Therapeutics. 334(1):106-115 (Jul. 2010).
[cited by applicant]
Life Technologies Corp. mMessage mMachine Kit (rev. F. Nov. 2011).
[cited by applicant]
Lin, Tongxiang et al. A Chemical Platform for Improved Induction of Human iPSCs. Nature Methods vol. 6,11: pp. 805-808 (2009).
[cited by applicant]
Liu et al.: A Small-Molecule Agonist of the Wnt Signaling Pathway. Angew. Chem. Int. Ed. 44: 1987-1990 (2005).
[cited by applicant]
Lonza: General protocol for nucleofection of adherent cell lines. (2008).
[cited by applicant]
Lu et al., “Defined culture conditions of human embryonic stem cells,” PNAS 103: 5688-5693 (2006).
[cited by applicant]
Ludwig et al. Derivation of human embryonic stem cells in defined conditions. Nat Biotechnol. 24(2):185-7 (Feb. 2006).
[cited by applicant]
Ludwig et al., “Feeder-independent culture of human embryonic stem cells,” Nat Methods 3:637-646 (2006).
[cited by applicant]
Mahfouz et al., “De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks,” PNAS 108(6): 2623-2628 (2011).
[cited by applicant]
Miller et al., “A TALE nuclease architecture for efficient genome editing,” Nature Biotechnology 29(2): 143-148 (2011).
[cited by applicant]
Miller et al., “An improved zinc-finger nuclease architecture for highly specific genome editing,” Nat. Biotechnot 25(7): 778-785 (2007).
[cited by applicant]
MIT Thesis Record, “Reprogramming human somatic cells to pluripotency using RNA,” (Matthew Angel, author) (2012).
[cited by applicant]
Moscou et al_, “A Simple Cipher Governs DNA Recognition by TAL Effectors,” Science 326: 1501 (2009).
[cited by applicant]
Mussolino, C, et al., “A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity”, Nucleic Acids Research, 2011, vol. 39, No. 1, pp. 9283-9293.
[cited by applicant]
NG et at, “A protocol describing the use of a recombinant protein-based, animal product-free medium (APEL) for human embryonic stem cell differentiation as spin embryoid bodies,” Nat. Protoc. 3: 768-776 (2008).
[cited by applicant]
Niu et al., (2008) Engineering Variants of the I-Scel Homing Endonuclease with Strand-specific and Site-specific DNA nicking Activity, Journal of Molecular Biology 382: 188-202 (2008).
[cited by applicant]
No Author, Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728 for HIV (Zinc-Finger), View of NCT00842634 on Feb. 11, 2009, ClinicalTrials.gov archive (Feb. 12, 2009) https://clinica…
[cited by applicant]
No Author, “mMessage mMachine Kit (Cat#AM1340, AM1344, AM1348)”, Instruction Manual, Jan. 4, 2007, pp. 1-34.
[cited by applicant]
Okita, keisuke et al. Generation of Germline-competent Induced Pluripotent Stem Cells. Nature vol. 448,7151: pp. 313-317 (2007).
[cited by applicant]
Patel et al.: Advances in reprogramming somatic cells to induced pluripotent stem cells. Stem Cell Rev 6(3):367-380 (2010).
[cited by applicant]
PCT/US2012/067966 International Preliminary Report on Patentability, 7 pages (Jun. 10, 2014).
[cited by applicant]
PCT/US2012/067966 International Search Report , 5 pages (Apr. 11, 2013).
[cited by applicant]
Plews et al., “Activation of pluripotency genes in human fibroblast cells by a novel mRNA based approach,” PLoS One 5:e14397 pp. 1-10 (2010).
[cited by applicant]
Porteus et al., “Gene targeting using zinc finger nucleases,” Nat. Biotechnol. 23(8): 967-973 (2005).
[cited by applicant]
Rossi et al., “Anti-inflammatory cyclopentenone prostaglandins are direct inhibitors of IkB kinase,” Nature 403: 103-108 (2000).
[cited by applicant]
Sander et al., “Targeted gene disruption in somatic zebrafish cells using engineered TALENs,” Author Manuscript, available in PMC on Feb. 5, 2012. Published in final edited form as: Nat Biotechnol. ; 29(8): 697-698 (201…
[cited by applicant]
Sanjana et al., “A transcription activator-like effector toolbox for genome engineering,” Nature Protocols 7(1): 171-192 (2012).
[cited by applicant]
Scheider et al., “An effective method for defatting albumin using resin columns,” Biochim. Biophys 221: 376-378 (1970).
[cited by applicant]
Schwartz et al., “Embryonic stem cell trials for macular degeneration: a preliminary report,” Lancet, pp. 1-8 (2012).
[cited by applicant]
Sharova, L.V, et al., “Database for mRNA Half-Life of 19 977 Genes Obtained by DNA Microarray Analysis of Pluripotent and Differentiating Mouse Embryonic Stem Cells”, DNA Research, Feb. 2009; 16(I):45-58.
[cited by applicant]
Shimizu et al., “Transformation by Wnt Family Proteins Correlates with Regulation of 13-Catenin,” Cell Growth & Differentiation 8: 1349-1358 (1997).
[cited by applicant]
Simões et al.: Human serum albumin enhances DNA transfection by lipoplexes and confers resistance to inhibition by serum. Biochim Biophys Acta. 1463:459-469 (2000).
[cited by applicant]
Soldner et al., “Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations,” Author Manuscript, available in PMC on Jul. 22, 2012. Published in final edited form as…
[cited by applicant]
Sugii, S. et al., “Human and Mouse Adipose-Derived Cells Support Feeder-Independent Induction of Pluripotent Stem Cells”, PNAS, 2010, vol. 107, No. 8, pp. 3558-3563.
[cited by applicant]
Takahashi et al., “Induction of pluripotent stem cells from adult human fibroblasts by defined factors,” Cell 131: 1-12 (2007).
[cited by applicant]
Takahashi, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. Nov. 30, 2007;131(5):861-72.
[cited by applicant]
Takahashi, et al., “Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors,” Cell 126: 1-14 (2006).
[cited by applicant]
Tesson et al.: Knockout rats generated by embryo microinjection of TALENs. Nature Biotechnology 29(8): 695-696 (2011).
[cited by applicant]
Urnov, F D, et al., Genome editing with engineered zinc finger nucleases Nature Reviews Genetics, Sep. 2010, vol. 11, pp. 636-646.
[cited by applicant]
U.S. Appl. No. 90/019,127 Order Granting Request for Ex Parte Reexamination dated Jan. 31, 2023.
[cited by applicant]
U.S. Appl. No. 90/019,127 Request for Ex Parte Examination of U.S. Pat. No. 10,662,410 dated Nov. 16, 2022.
[cited by applicant]
U.S. Appl. No. 90/019,128 Order Granting Request for Ex Parte Reexamination dated Jan. 31, 2023.
[cited by applicant]
U.S. Appl. No. 90/019,128 Request for Ex Parte Examination of U.S. Pat. No. 10,829,738 dated Nov. 16, 2022.
[cited by applicant]
U.S. Appl. No. 90/019,129 Order Granting Request for Ex Parte Reexamination dated Jan. 31, 2023.
[cited by applicant]
U.S. Appl. No. 90/019,129 Request for Ex Parte Examination of U.S. Pat. No. 10,982,229 dated Nov. 16, 2022.
[cited by applicant]
U.S. Ex-Parte Re Exam U.S. Appl. No. 90/019,127 Office Action dated Aug. 29, 2023.
[cited by applicant]
U.S. Ex-Parte Re Exam U.S. Appl. No. 90/019,128 Office Action dated Sep. 8, 2023.
[cited by applicant]
U.S. Ex-Parte Re Exam U.S. Appl. No. 90/019,129 Office Action dated Sep. 8, 2023.
[cited by applicant]
U.S. Appl. No. 16/567,059 Office Action dated Mar. 11, 2022.
[cited by applicant]
U.S. Appl. No. 16/913,306 Notice of Allowance dated May 15, 2023.
[cited by applicant]
U.S. Appl. No. 16/913,306 Office Action dated Nov. 25, 2022.
[cited by applicant]
U.S. Appl. No. 16/913,315 Office Action dated Nov. 25, 2022.
[cited by applicant]
U.S. Appl. No. 17/821,298 Office Action dated Mar. 28, 2024.
[cited by applicant]
Van De Parre TJ, et al., “mRNA but not plasmid DNA is efficiently transfected in murine J774A.1 macrophages”, Biochem Biophys Res Commun., 2005, 327(I):356-60.
[cited by applicant]
Van Tendeloo VF, et al., “Highly efficient gene delivery by mRNA electroporation in human hematopoietic cells: superiority to lipofection and passive pulsing of mRNA and to electroporation of plasmid cDNA for tumor anti…
[cited by applicant]
Warren et al., “Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA,” Cell. Stem Cell 7: 1-13 (2010).
[cited by applicant]
Watanabe, Kiichi et al. A ROCK Inhibitor Permits Survival of Dissociated Human Embryonic Stem Cells. Nature Biotechnology vol. 25,6: pp. 681-686 (2007).
[cited by applicant]
Wernig et al., “In vitro reprogramming of fibroblasts into a pluripotent ES-cell-like state,” Nature 448: 317-324 (2007).
[cited by applicant]
Wiehe, JM, et al., “mRNA-mediated gene delivery into human progenitor cells promotes highly efficient protein expression”, J Cell Mol Med., 2007, vol. 3, pp. 521-530.
[cited by applicant]
Wood et al., “Targeted Genome Editing Across Species Using ZFNs and TALENs,” Science 333: 307 (2011).
[cited by applicant]
Xeno-Free System for hESC & hiPSC. Facilitating the Shift from Stem Cell Research to Clinical Applications. 12 pages, Biological Industries Catalog (Stem Cell Products) (2011).
[cited by applicant]
Xie et al., “Newly expressed proteins of mouse embryonic fibroblasts irradiated to be inactive,” Biochem. Biophys. Res Commun. 315: 581-588 (2004).
[cited by applicant]
Yakubov et al., “Reprogramming of human fibroblasts to pluripotent stem cells using mRNA of four transcription factors,” Biochem Biophys Res Commun. 394: 189-193 (2010).
[cited by applicant]
You et al., “Wnt signaling promotes oncogenic transformation by inhibiting c-Myc-induced apoptosis,” The Journal of well Biology 157(3): 429-440 (2002).
[cited by applicant]
Young et al., “Background Mutations in Parental Cells Account for Most of the Genetic Heterogeneity of Induced Pluripotent Stem Cells,” Cell Stem Cell 10: 570-582 (2012).
[cited by applicant]
Yu et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 318:1917-1920 (2007).
[cited by applicant]
Zhou et al., “Generation of Induced Pluripotent Stem Cells Using Recombinant Proteins,” Cell Stem Cell 4: 1-4 (2009).
[cited by applicant]