Granted Patent
B2
US 12,202,860 · App. 18/130,375 · Granted Jan 21, 2025
Methods and products for genetic engineering
Inventors:
Théophile Ohlmann (Tassin la Demi-Lune, FR); Philippe Mangeot (Lyons, FR); Emiliano Ricci (Lyons, FR)
Assignees:
Institut National De La Sante Et De La Recherche Medicale (INSERM); Centre National De La Recherche Scientifique (CNRS); Ecole Normale Superieure De Lyon; Universite Claude Bernard Lyon 1
C07K14/005C12N7/00C12N9/22C12N15/102C12N15/113C07K2319/00C07K2319/43C07K2319/50C12N2310/20C12N2740/10023C12N2740/10042C12N2740/13023C12N2740/13042C12N2800/80
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Abstract
The present invention relates to a virus-derived particle comprising one or more Cas protein(s), as well as to kits and methods using the same for altering a target nucleic acid.
Claims (36)
1. A retrovirus-derived particle comprising one or more Cas protein(s), wherein the one or more Cas protein(s) is inside of said particle as a fusion protein between (i) a retroviral gag protein and (ii) the one or more Cas protein(s), wherein the one or more Cas protein(s) is complexed with one or more CRISPR-Cas guide RNA(s).
2. The retrovirus-derived particle of claim 1 , wherein the Cas protein is Cas9 or a homolog or a derivative thereof.
3. The retrovirus-derived particle of claim 1 , wherein the Cas protein is Cas9.
4. The retrovirus-derived particle of claim 3 , wherein the Cas9 is from Streptococcus pyogenes (spCas9).
5. The retrovirus-derived particle of claim 1 , wherein the Cas protein is present as a cleavable fusion protein comprising a proteolysis cleavage site located between the retroviral gag protein and the Cas protein.
6. The retrovirus-derived particle of claim 3 , wherein the Cas protein is present as a cleavable fusion protein comprising a proteolysis cleavage site located between the retroviral gag protein and the Cas protein.
7. The retrovirus-derived particle of claim 1 , which is a lentivirus-derived particle.
8. The retrovirus-derived particle of claim 3 , which is a lentivirus-derived particle.
9. The retrovirus-derived particle of claim 1 , wherein the retrovirus-derived particle is substantially devoid of any nucleic acid encoding a viral protein.
10. The retrovirus-derived particle of claim 1 , wherein the retrovirus-derived particle is devoid of any nucleic acid encoding a viral protein.
11. The retrovirus-derived particle of claim 1 , wherein the CRISPR-Cas guide RNA(s) comprise:
a first CRISPR-Cas guide RNA that hybridizes with a first target sequence of a target nucleic acid, and
a second CRISPR-Cas guide RNA that hybridizes with a second target sequence of the target nucleic acid.
12. The retrovirus-derived particle of claim 1 , wherein the retrovirus-derived particle is selected from the group consisting of Moloney murine leukemia virus-derived vector particles, Bovine immunodeficiency virus-derived particles, Simian immunodeficiency virus-derived vector particles, Feline immunodeficiency virus-derived vector particles, Human immunodeficiency virus-derived vector particles, Equine infection anemia virus-derived vector particles, Caprine arthritis encephalitis virus-derived vector particles, and Baboon endogenous virus-derived vector particles.
13. The retrovirus-derived particle of claim 12 , wherein the retrovirus-derived particle is a Moloney murine leukemia virus-derived vector particle.
14. The retrovirus-derived particle of claim 12 , where the retrovirus-derived particle is a Human immunodeficiency virus-derived vector particle.
15. The retrovirus-derived particle of claim 12 , where the retrovirus-derived particle is a Baboon endogenous virus-derived vector particle.
16. The retrovirus-derived particle of claim 5 , which is a lentivirus-derived particle.
17. The retrovirus-derived particle of claim 1 , wherein the retrovirus-derived particle further comprises a viral protease.
18. The retrovirus-derived particle of claim 1 , wherein the retrovirus-derived particle are devoid of any viral protease.
19. A composition, comprising at least one retrovirus-derived particle of claim 1 .
20. The composition of claim 19 , further comprising one or more transduction helper compounds.
21. A composition, comprising at least one retrovirus-derived particle of claim 3 .
22. The composition of claim 21 , further comprising one or more transduction helper compounds.
23. An isolated cell line for producing the retrovirus-derived particle of claim 1 , comprising:
one or more nucleic acids encoding proteins required for forming the retrovirus-derived particle of claim 1 ,
a nucleic acid comprising an expression cassette encoding a GAG-Cas fusion protein, and
nucleic acid(s) encoding one or more CRISPR guide RNA(s).
24. The isolated cell line of claim 23 , wherein the Cas protein is Cas9.
25. The isolated cell line of claim 23 , further comprising a nucleic acid comprising an expression cassette encoding a viral protease.
26. An in vitro or ex vivo method for altering, in at least one eukaryotic cell, a target nucleic acid comprising at least one target sequence, comprising the steps of:
a) contacting the at least one eukaryotic cell with one or more retrovirus-derived particles of claim 1 , wherein the step of contacting is performed under conditions which permit the retrovirus-derived particles to infect the at least one eukaryotic cell; and
b) collecting eukaryotic cells having an altered target nucleic acid.
27. An in vitro or ex vivo method for altering, in at least one eukaryotic cell, a target nucleic acid comprising at least one target sequence, comprising the steps of:
a) contacting the at least one eukaryotic cell with one or more retrovirus-derived particles of claim 3 , wherein the step of contacting is performed under conditions which permit the retrovirus-derived particles to infect the at least one eukaryotic cell; and
b) collecting eukaryotic cells having an altered target nucleic acid.
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Apr 5, 2023
From: OHLMANN, THÉOPHILE; MANGEOT, PHILIPPE; RICCI, EMILIANO
To: INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS); ECOLE NORMALE SUPERIEURE DE LYON; UNIVERSITE CLAUDE BERNARD LYON 1
Reel/Frame 063229/0823 →
Priority Claims (1)
EP 15306678
· Oct 20, 2015
· regional
Continuity (4)
Continuation
15769534
Related Publication
20240228546A9
· Jul 11, 2024
References Cited (148)
US 6060064A
· Elizabeth et al.
· 2000
[cited by applicant]
US 6204059B1
· Samulski et al.
· 2001
[cited by applicant]
US 20110262483A1
· Haynes et al.
· 2011
[cited by applicant]
US 20120269840A1
· Barnett et al.
· 2012
[cited by applicant]
US 20190010518A1
· Quake et al.
· 2019
[cited by applicant]
WO WO1992012237
· 1992
[cited by applicant]
WO WO1995022617
· 1995
[cited by applicant]
WO WO2010040023
· 2010
[cited by applicant]
WO WO2013068847
· 2013
[cited by applicant]
WO WO2013163628
· 2013
[cited by applicant]
WO WO2013176772
· 2013
[cited by applicant]
WO WO2014093595
· 2014
[cited by applicant]
WO WO2014131833
· 2014
[cited by applicant]
WO WO2014200659
· 2014
[cited by applicant]
WO WO2015089247
· 2015
[cited by applicant]
WO WO2015089486
· 2015
[cited by applicant]
WO WO2016073433
· 2016
[cited by applicant]
WO WO2017001570
· 2017
[cited by applicant]
WO WO2017106822
· 2017
[cited by applicant]
Mazurov D, Ramadan L, Kruglova N. Packaging and Uncoating of CRISPR/Cas Ribonucleoproteins for Efficient Gene Editing with Viral and Non-Viral Extracellular Nanoparticles. Viruses. Mar. 6, 2023;15(3):690. (Year: 2023).
[cited by examiner]
Abe et al., “In vitro cell-free conversion of noninfectious moloney retrovirus particles to an infectious form by the addition of the vesicular stomatitis virus surrogate envelope G protein,” J. Virol. 72(8):6356-6361 (…
[cited by applicant]
Aliyari et al., “RNA-based viral immunity initiated by the Dicer family of host immune receptors,” Immunol Rev. (2009) 227(1): 176-88.
[cited by applicant]
Anastasov et al., Lentiviral vectors and exosomes as gene and protein delivery tools, in Methods in Molecular Biology, (2016) 1448: 49-61.
[cited by applicant]
Aoki et al., “Protein transduction ny pseudotyped lentivirus-like nanoparticles,” Gene Therapy (2011) 1-6.
[cited by applicant]
Brandler et al. “Recombinant vector derived from live attenuated measles virus: potential for flavivirus vaccines,” Comp Immunol Microbiol Infect Dis. (2008) 31 (No. 2-3): 271-291.
[cited by applicant]
Brown et al., “Assembly of hybrid bacteriophage Qbeta virus-like particles,” Biochemistry. (2009) 48(47):11155-11157.
[cited by applicant]
Cai et al., “Targeted genome editing by lentiviral protein transduction of ZFN and Cas9 proteins,” Selected oral presentation (2014) A31:OR021.
[cited by applicant]
Cai et al., “Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases.” (2014) Elife sciences publications Ltd. 3: 1-19.
[cited by applicant]
Cass et al., “The role of Cas8 in type I Crispr interference,” Biosci Rep. (2015) 5;35(3):e00197. (doi:10.1042/BSR20150043).
[cited by applicant]
Cervera et al., “Generation of HIV-1 Gag VLPs by transient transfection of HEK 293 suspension cell cultures using an optimized animal-derived component free medium,” J Biotechnol. (2013) 166(4): 152-65.
[cited by applicant]
Choi JG, Dang Y, Abraham S, Ma H, Zhang J, Guo H, Cai Y, Mikkelsen JG, Wu H, Shankar P, Manjunath N. Lentivirus pre-packed with Cas9 protein for safer gene editing. Gene Ther. Jul. 2016 ;23(7) :627-33. doi: 10.1038/gt.2…
[cited by applicant]
Chylinski et al. “The tracrRNA and Cas9 families of type II CRISPR-Cas immunity systems,” RNA Biol. May 1, 2013; 10(5): 726-737.
[cited by applicant]
Chylinski et al., “Classification and evolution of type II CRISPR-Cas systems,” Nucleic Acids Res. (2014) 42(10):6091-105.
[cited by applicant]
Cong et al., “Multiplex Genome Engineering Using CRISPR/Cas Systems,” Science (2013) 339(6121): 819-823.
[cited by applicant]
Cronin et al., “Altering the tropism of lentiviral vectors through pseudotyping,” Curr Gene Ther. (2005) 5(4):387-98.
[cited by applicant]
Dale et al.: “Tracking and quantitation of fluorescent HIV during cell-cell transmission”, Methods. Jan. 2011; 53(1): 20-26. DOI:10.1016/J.YMETH.2010.06.018.
[cited by applicant]
DiCarlo et al., “Genome engineering in
[cited by applicant]
Esvelt et al., “Orthogonal Cas9 proteins for RNA-guided gene regulation and editing,” Nat Methods. (2013) 10(11):1116-21. doi: 10.1038/nmeth.2681.
[cited by applicant]
Fabre et al., “CRISPR is an optimal target for the design of specific PCR assays for
[cited by applicant]
Farboud et al. “Dramatic Enhancement of Genome Editing by CRISPR/Cas9 Through Improved Guide RNA Design,” Genetics, (2015) 199(4); 959-971, (doi 10.1534/genetics.115.175166).
[cited by applicant]
Fenard et al., “Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of human hematopoietic stem cells,” Mol Ther Nucleic Acids. May 7, 2013;2(5):e90.
[cited by applicant]
Fontana et al., “Rabies virus-like particles expressed in HEK293 cells,” Vaccine. (2014) 32(24); 2799-804.
[cited by applicant]
Fu A, Tang R, Hardie J, Farkas ME, Rotella VM. Promises and pitfalls of intracellular delivery of proteins. Bioconjug Chem. Sep. 17, 2014;25(9):1602-8. Epub Sep. 2, 2014.
[cited by applicant]
Garrone et al.: “A prime-boost Strategy Using Virus-Like Particles Pseudotyped for HGV Proteins Triggers Broadly Neutralizing Antibodies in Macaques”, Science Translation Medicine. Aug. 3, 2011. vol. 3 Issue.
[cited by applicant]
Gasiunas et al., “Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria,” Proc Natl Acad Sci USA. (2012) 109(39):E2579-86.
[cited by applicant]
Girard-Gagnepain et al., “Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs,” Blood.(2014) 124 (8): 1221-1231.
[cited by applicant]
Gong et al., “Mechanism of nonhomologous end-joining in mycobacteria: a low-fidelity repair system driven by Ku, ligase D and ligase C,” Nat Struct Mol Biol. (2005) 12(4):304-12.
[cited by applicant]
Gori JL, Hsu PD, Maeder ML, Shen S, Welstead GG, Bumcrot D. Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy. Hum Gene Ther. Jul. 2015;26(7):443-51.
[cited by applicant]
Gratz et al., “Highly specific and efficient CRISPR/Cas9-catalyzed homology-directed repair in
[cited by applicant]
Guibingua et al., “Cell surface heparan sulfate is a receptor for attachment of envelope protein-free retrovirus-like particles and VSV-G pseudotyped MLV-derived retrovirus vectors to target cells,” Mol Ther. (2002) 5(5…
[cited by applicant]
Guo et al., Efficient RNA/Cas9-mediated genome editing in Xenopus tropicalis,' 2014, Development. (2014) 141(3):707-14.
[cited by applicant]
Hackett et al., Delivering the second revolution in seite-specififc nucleases, Elife 2014 e02904.
[cited by applicant]
Hai et al., “One-step generation of knockout pigs by zygote injection of CRISPR/Cas system,” (2014) Cell Res. 24: 372-375. doi: 10.1038/cr.2014.11.
[cited by applicant]
Heler et al., “Cas9 specifies functional viral targets during CRISPR-Cas adaptation,” Nature. (2015) 519(7542): 199-202.
[cited by applicant]
Herbst-Kralovetz et al., “Norwalk virus-like particles as vaccines,” Expert Rev Vaccines. (2010) 9(3): 299-307.
[cited by applicant]
Hill BD, Zak A, Khera E, Wen F. Engineering Virus-like Particles for Antigen and Drug Delivery. Curr Protein Pept Sci. 2018;19(1): 112-127.
[cited by applicant]
Hochstrasser et al., “CasA mediates Cas3-catalyzed target degradation during CRISPR RNA-guided interference,” PNAS (2014) 111(18):6618-6623.
[cited by applicant]
Hong et al., “Novel recombinant hepatitis B virus vectors efficiently deliver protein and RNA encoding genes into primary hepatocytes,” J Virol. (2013) 87(12):6615-24.
[cited by applicant]
Horii et al: “Efficient generation of knockin mice by CRISPR/Cas system”, Experimental Animals vol. 64, abstract, 2015.
[cited by applicant]
Hwang et al., “Heritable and precise zebrafish genome editing using a CRISPR-Cas system,” PLoS One. (2013) 8(7):e68708.
[cited by applicant]
Jalaguier et al., “Efficient production of HIV-1 virus-like particles from a mammalian expression vector requires the N-terminal capsid domain,” PLoS One. (2011) 6(11):e28314.
[cited by applicant]
Jao et al., “Efficient multiplex biallelic zebrafish genome editing using a CRISPR nuclease system,” Proc Natl Acad Sci U S A. (2013) 110(34):13904-13909.
[cited by applicant]
Jinek et al., “A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity,” Science. (2012) 337(6096) :816-21.
[cited by applicant]
Jinek et al., “RNA-programmed genome editing in human cells,” Elife. (2013) 2:e00471. doi: 10.7554/eLife.00471.
[cited by applicant]
Johnston et al., “High-throughput screening identifies compounds that enhance lentiviral transduction,” Gene Ther. (2014) 21(12): 1008-20.
[cited by applicant]
Kaczmarczyk SJ, Sitaraman K, Young HA, Hughes SH, Chatterjee DK. Protein delivery using engineered virus-like particles. Proc Natl Acad Sci U SA. Oct. 11, 2011; 108(41): 16998-7003. Epub Sep. 26, 2011.
[cited by applicant]
Kajigaya et al., “Self-assembled B19 parvovirus capsids, produced in a baculovirus system, are antigenically and immunogenically similar to native virions,” Proc Natl Acad Sci USA. (1991) 88(11):4646-50.
[cited by applicant]
Kang et al., “Chimeric rabies virus-like particles containing membrane-anchored GM-CSF enhances the immune response against rabies virus,” Viruses. (2015) 7(3): 1134-52. doi: 10.3390/v7031134.
[cited by applicant]
Kieusseian et al., “Expression of Pitx2 in stromal cells is required for normal hematopoiesis,” Blood. (2006) 107(2):492-500.
[cited by applicant]
Kim et al., “Highly efficient GENOME RNA-guided Research, genome vol. 24, editing pages in human 1012-1019, cells via 2014 delivery of purified Cas9 ribonucleoproteins,” Genome Research (2014) 24:1012-1019.
[cited by applicant]
Kirnbauer et al., “Virus-like particles of bovine papillomavirus type 4 in prophylactic and therapeutic immunization,” Virology. (1996) 219(1):37-44.
[cited by applicant]
Kushnir et al., “Virus-like particles as a highly efficient vaccine platform: diversity of targets and production systems and advances in clinical development,” Vaccine. (2012) 31(1):58-83.
[cited by applicant]
Lafountaine Justin S et al: “Delivery and therapeutic applications of gene editing technologies ZFNs, TALENs, and CRISPR/Cas9”, International Journal of Pharmaceutics, vol. 494, No. 1, 2015 (Aug. 13, 2015.
[cited by applicant]
Latham et al., “Formation of wild-type and chimeric influenza virus-like particles following simultaneous expression of only four structural proteins,” J Virol. (2001) 75(13):6154-65.
[cited by applicant]
Li et al., “Expression and self-assembly of empty virus-like particles of hepatitis E virus,” J Virol. (1997) 71(10):7207-13.
[cited by applicant]
Lin et al., “Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery,” Elife. (2014) 15;3:e04766.
[cited by applicant]
Lombardo et al., “TLR4-mediated survival of macrophages is MyD88 dependent and requires TNF-alpha autocrine signalling,” J Immunol. (2007) 178(6): 3731-3739.
[cited by applicant]
Ludwig et al., “Virus-like particles-universal molecular toolboxes,” Curr Opin Biotechnol. (2007) 18(6):537-45.
[cited by applicant]
Ma et al., “A guide RNA sequence design platform for the CRISPR/Cas9 system for model organism genomes,” Biomed Res Int. (2013) ;2013:270805. doi: 10.1155/2013/270805.
[cited by applicant]
Ma et al., “Generating rats with conditional alleles using CRISPR/Cas9,” Cell Res. (2014) 24(1):122-5.
[cited by applicant]
Maeder et al., “CRISPR RNA-guided activation of endogenous human genes,” Nat Methods. (2013) 10(10):977-9.
[cited by applicant]
Maetzig et al., “Retroviral protein transfer: falling apart to make an impact,” Curr Gene Ther. (2012) 12(5):389-409.
[cited by applicant]
Makarova et al., “Unification of Cas protein families and a simple scenario for the origin and evolution of CRISPR-Cas systems,” Biol Direct. 2011; 6: 38.
[cited by applicant]
Mali et al., “RNA-guided human genome engineering via Cas9,” Science. (2013) 339(6121): 823-826.
[cited by applicant]
Mangeot et al. (2011, Mol Ther J Am Soc Gene Ther, vol. 19: 1656-1666.
[cited by applicant]
Mangeot et al., “A universal transgene silencing method based on RNA interference,” Nucleic Acids Res. (2004) 32(12):e102.
[cited by applicant]
Mangeot et al., “Development of minimal lentivirus vectors derived from simian immunodeficiency virus (SIVmac251) and their use for gene transfer into human dendritic cells,” J Virol. (2000) 74(18): 8307-8315.
[cited by applicant]
Markowitz et al., “A safe packaging line for gene transfer: separating viral genes on two different plasmids,” J. Virol. (1988) 62(4): 1120.
[cited by applicant]
Mashiko et al., “Feasibility for a large scale mouse mutagenesis by injecting CRISPR/Cas plasmid into zygotes,” Dev Growth Differ. (2014) 56(1):122-9.
[cited by applicant]
Mateu MG. Assembly, Engineering and Applications of Virus-Based Protein Nanoparticles. Adv Exp Med Biol. 2016;940:83-120. Review. PubMed PMID: 27677510.
[cited by applicant]
Mselli-Lakhal et al., “Gene transfer system derived from the caprine arthritis-encephalitis lentivirus,” J Virol Methods. (2006) 136(1-2):177-184.
[cited by applicant]
Murawski et al., “Newcastle disease virus-like particles containing respiratory syncytial virus G protein induced protection in BALB/c mice, with no evidence of immunopathology,” J Virol. (2010) 84(2):1110-23.
[cited by applicant]
Naskalska et al., “Virus Like Particles as Immunogens and Universal Nanocarriers, ” Pol J Microbiol. (2015) 64(1): 3-13.
[cited by applicant]
Negre et al., “Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells,” (2000) Gene Ther. 7(19): 1613-1623.
[cited by applicant]
Niu et al., “Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos,” Cell. (2014) 156(4): 836-43.
[cited by applicant]
O'Connell et al., “Programmable RNA recognition and cleavage by CRISPR/Cas9,” Nature. (2014) 516(7530): 263-266.
[cited by applicant]
Ogasawara et al., “Recombinant viral-like particles of parvovirus B19 as antigen carriers of anthrax protective antigen,” In Vivo. (2006) 20(3): 319-24.
[cited by applicant]
Oji et al: “Complex genome editing in mouse ES cells using the CRISPR/Cas system”, Experimental Animals.
[cited by applicant]
Okimoto et al., “VSV-G envelope glycoprotein forms complexes with plasmid DNA and MLV retrovirus-like particles in cell-free conditions and enhances DNA transfection,” Mol Ther. (2001) 4(3): 232-8.
[cited by applicant]
Olsen, “Gene transfer vectors derived from equine infectious anemia virus,” Gene Ther. (1998) 5(11):1481-1487.
[cited by applicant]
Overballe-Petersen et al., “Bacterial natural transformation by highly fragmented and damaged DNA,” Proc Natl Acad Sci USA. (2013) 110(49):19860-19865.
[cited by applicant]
Peifang et al., “Enhanced activation of human T cell clones specific for virus-like particles expressing the HIV V3 loop in the presence of HIV V3 loop-specific polyclonal antibodies,” Clin Exp Immunol. (1994) 97(3): 36…
[cited by applicant]
Qazi et al.: “Programmed Self-Assembly of an Active P22-Cas9 Nanocarrier System”, Molecular Pharmaceutics, vol. 13, No. 3, Mar. 7, 2016 (Mar. 7, 2016), pp. 1191-1196, XP055268807.
[cited by applicant]
Qi et al., “Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression,” Cell. (2013) 152(5): 1173-83.
[cited by applicant]
Quan et al., “Influenza M1 VLPs containing neuraminidase induce heterosubtypic cross-protection,” Virology. (2012) 430(2): 127-35.
[cited by applicant]
Ramakrishna et al: “Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide Rna”, Genome Research, vol. 24, pp. 1020-1027, Apr. 2, 2014.
[cited by applicant]
Ran et al., “Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity,” Cell. (2013) 154(6): 1380-9.
[cited by applicant]
Rasmussen et al., “Characterization of virus-like particles produced by a recombinant baculovirus containing the gag gene of the bovine immunodeficiency-like virus,” Virology. (1990) 178(2): 435-51.
[cited by applicant]
Rohovie MJ, Nagasawa M, Swartz JR. Virus-like particles: Next-generation nanoparticles for targeted therapeutic delivery. Bioeng Transl Med. Jan. 19, 2017;2(1):43-57.
[cited by applicant]
Ruffing et al., “Assembly of viruslike particles by recombinant structural proteins of adeno-associated virus type 2 in insect cells,” J Virol. (1992) 66(12):6922-6930.
[cited by applicant]
Saenz et al., “Production and harvest of feline immunodeficiency virus-based lentiviral vector from cells grown in T75 tissue-culture flasks,” Cold Spring Harb Protoc. (2012) 2012(1):124-125.
[cited by applicant]
Saenz et al., “Feline immunodeficiency virus-based lentiviral vectors,” Cold Spring Harb Protoc. (2012) 2012(1):71-6.
[cited by applicant]
Saenz et al., “Production, harvest, and concentration of feline immunodeficiency virus- based lentiviral vector from cells grown in CF10 or CF2 devices,” Cold Spring Harb Protoc. (2012) 2012(1): 118-23.
[cited by applicant]
Sakuma et al., Multiplex genome engineering in human cells using all in one CRISPR/Cas9 vector system. Sci Rep. 4: 5400, 2014.
[cited by applicant]
Samulski et al., “Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression,” J Virol. (1989) 63(9): 3822-8.
[cited by applicant]
Sanjana et al., “Improved vectors and genome-wide libraries for CRISPR screening,” Nat Methods. (2014) 11(8):783-784.
[cited by applicant]
Shalem et al., “Genome-scale CRISPR-Cas9 knockout screening in human cells,” Science. (2014) 343(6166): 84-87.
[cited by applicant]
Sharma et al., “Noninfectious virus-like particles produced by Moloney murine leukemia virus-based retrovirus packaging cells deficient in viral envelope become infectious in the presence of lipofection reagents,” Proc …
[cited by applicant]
Sinkunas et al., “Cas3 is a single-stranded DNA nuclease and ATP-dependent helicase in the CRISPR/Cas immune system,” EMBO J. (2011) 30(7):1335-42.
[cited by applicant]
Skipper et al., “Delivering the goods for genome engineering and editing.” Hum Gene Therapy. (2015) 26(8):486-497.
[cited by applicant]
Sun et al: 11 Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing11 , Angewandte Chemie International Edition, vol. 54, No. 41, Oct. 5, 2015 (Oct. 5, 2015).
[cited by applicant]
Takahara et al., “A new retrovirus packaging cell for gene transfer constructed from amplified long terminal repeat-free chimeric proviral genes,” J Virol. (1992) 66(6): 3725-3732.
[cited by applicant]
Tomé-Amat et al., “Secreted production of assembled Norovirus virus-like particles from Pichia pastoris,” Microb Cell Fact. (2014) 13:134-142.
[cited by applicant]
Voelkel C, Galla M, Maetzig T, Warlich E, Kuehle J, Zychlinski D, Bode J, Cantz T, Schambach A, Baum C. Protein transduction from retroviral Gag precursors. Proc Natl Acad Sci U SA. Apr. 27, 2010; 107(17):7805-10. doi: …
[cited by applicant]
Wagner et al., “Transfer of genes into embryonal carcinoma cells by retrovirus infection: efficient expression from an internal promoter,” EMBO J. (1985) 4(3):663-6.
[cited by applicant]
Walpita et al., “Mammalian Cell-Derived Respiratory Syncytial Virus-Like Particles Protect the Lower as well as the Upper Respiratory Tract,” PLoS One. (2015) 10(7): e0130755.
[cited by applicant]
Wang et al., “One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering,” Cell. (2013) 153(4):910-918.
[cited by applicant]
Wang et al., “Virus-like particles for the prevention of human papillomavirus-associated malignancies,” Expert Rev Vaccines. (2013) 12(2):129-41.
[cited by applicant]
Yang et al., “Effective gene targeting in rabbits using RNA-guided Cas9 nucleases,” J Mol Cell Biol. (2014) 6(1):97-99.
[cited by applicant]
Yang et al., “HIV-1 virus-like particles produced by stably transfected
[cited by applicant]
Yee et al., “A general method for the generation of high-titer, pantropic retroviral vectors: highly efficient infection of primary hepatocytes,” Proc Natl Acad Sci USA. (1994) 91(20):9564-9568.
[cited by applicant]
Yee et al., “Generation of high-titer pseudotyped retroviral vectors with very broad host range,” Methods Cell Biol. (1994);43 Pt A: 99-112.
[cited by applicant]
Zeltins, Andris. “Construction and characterization of virus-like particles: a review.” Mol Biotechnol. (2013) 53(1): 92-107.
[cited by applicant]
Zetsche et al., “Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system,” Cell. (2015) 163(3):759-71. http://dx.doi.org/10.1016/j.cell.2015.09.038.
[cited by applicant]
Zhao et al., “BPV1 E2 protein enhances packaging of full-length plasmid DNA in BPV1 pseudovirions,” Virology. (2000) 272(2): 382-93.
[cited by applicant]
Zhu et al., “CRISPRseek: a bioconductor package to identify target-specific guide RNAs for CRISPR-Cas9 genome-editing systems,” PLoS One. (2014) 9(9):e108424.
[cited by applicant]
Zuris, John A., et al. “Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo.” Nature biotechnology 33.1 (2015): 73-80.
[cited by applicant]
Zuris et al., “Efficient delivery of genome-editing proteins in vitro and in vivo,” Nat Biotechnol (2015) 33(1):73-80.
[cited by applicant]
Kabadi et al., “Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector,” Nucleic Acids Res. (2014) 42(19):e147 (11 pages).
[cited by applicant]
Horvath et al. (Mar. 12, 2013), “RNA-guided genome editing à la carte,”
[cited by applicant]
Richter et al. (Jul. 12, 2013), “Exploiting CRISPR/Cas: Interference Mechanisms and Applications,”
[cited by applicant]
Shalem et al., “Genome-scale CRISPR-Cas9 knockout screening in human cells,” Science. (2014) 343(6166): Supplementary Information (78 pages).
[cited by applicant]
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