pX601-AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA Citations (129)
Originally described in: In vivo genome editing using Staphylococcus aureus Cas9.Ran FA, Cong L, Yan WX, Scott DA, Gootenberg JS, Kriz AJ, Zetsche B, Shalem O, Wu X, Makarova KS, Koonin EV, Sharp PA, Zhang F Nature. 2015 Apr 1. doi: 10.1038/nature14299. PubMed Journal
Articles Citing pX601-AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA
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| Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study. Kaminski R, Bella R, Yin C, Otte J, Ferrante P, Gendelman HE, Li H, Booze R, Gordon J, Hu W, Khalili K. Gene Ther. 2016 May 19. doi: 10.1038/gt.2016.41. PubMed |
| Expanding CRISPR/Cas9 Genome Editing Capacity in Zebrafish Using SaCas9. Feng Y, Chen C, Han Y, Chen Z, Lu X, Liang F, Li S, Qin W, Lin S. G3 (Bethesda). 2016 Jun 17. pii: g3.116.031914. doi: 10.1534/g3.116.031914. PubMed |
Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and beta-thalassemia.
Ye L, Wang J, Tan Y, Beyer AI, Xie F, Muench MO, Kan YW.
Proc Natl Acad Sci U S A. 2016 Sep 20;113(38):10661-5. doi: 10.1073/pnas.1612075113. Epub 2016 Sep 6.
PubMed
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Integrin-YAP/TAZ-JNK cascade mediates atheroprotective effect of unidirectional shear flow.
Wang L, Luo JY, Li B, Tian XY, Chen LJ, Huang Y, Liu J, Deng D, Lau CW, Wan S, Ai D, Mak KK, Tong KK, Kwan KM, Wang N, Chiu JJ, Zhu Y, Huang Y.
Nature. 2016 Dec 7. doi: 10.1038/nature20602.
PubMed
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| Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia. Ouellet DL, Cherif K, Rousseau J, Tremblay JP. Gene Ther. 2017 May;24(5):265-274. doi: 10.1038/gt.2016.89. Epub 2016 Dec 26. PubMed |
Re-engineered RNA-Guided FokI-Nucleases for Improved Genome Editing in Human Cells.
Havlicek S, Shen Y, Alpagu Y, Bruntraeger MB, Zufir NB, Phuah ZY, Fu Z, Dunn NR, Stanton LW.
Mol Ther. 2017 Feb 1;25(2):342-355. doi: 10.1016/j.ymthe.2016.11.007.
PubMed
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| Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Bengtsson NE, Hall JK, Odom GL, Phelps MP, Andrus CR, Hawkins RD, Hauschka SD, Chamberlain JR, Chamberlain JS. Nat Commun. 2017 Feb 14;8:14454. doi: 10.1038/ncomms14454. PubMed |
In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni.
Kim E, Koo T, Park SW, Kim D, Kim K, Cho HY, Song DW, Lee KJ, Jung MH, Kim S, Kim JH, Kim JH, Kim JS.
Nat Commun. 2017 Feb 21;8:14500. doi: 10.1038/ncomms14500.
PubMed
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| In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models. Yin C, Zhang T, Qu X, Zhang Y, Putatunda R, Xiao X, Li F, Xiao W, Zhao H, Dai S, Qin X, Mo X, Young WB, Khalili K, Hu W. Mol Ther. 2017 May 3;25(5):1168-1186. doi: 10.1016/j.ymthe.2017.03.012. Epub 2017 Mar 30. PubMed |
| No evidence of genome editing activity from Natronobacterium gregoryi Argonaute (NgAgo) in human cells. Javidi-Parsijani P, Niu G, Davis M, Lu P, Atala A, Lu B. PLoS One. 2017 May 11;12(5):e0177444. doi: 10.1371/journal.pone.0177444. eCollection 2017. PubMed |
| CRISPR/Cas9-loxP-Mediated Gene Editing as a Novel Site-Specific Genetic Manipulation Tool. Yang F, Liu C, Chen D, Tu M, Xie H, Sun H, Ge X, Tang L, Li J, Zheng J, Song Z, Qu J, Gu F. Mol Ther Nucleic Acids. 2017 Jun 16;7:378-386. doi: 10.1016/j.omtn.2017.04.018. Epub 2017 Apr 25. PubMed |
| Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism. Kemaladewi DU, Maino E, Hyatt E, Hou H, Ding M, Place KM, Zhu X, Bassi P, Baghestani Z, Deshwar AG, Merico D, Xiong HY, Frey BJ, Wilson MD, Ivakine EA, Cohn RD. Nat Med. 2017 Aug;23(8):984-989. doi: 10.1038/nm.4367. Epub 2017 Jul 17. PubMed |
Generation of a SOX2 reporter human induced pluripotent stem cell line using CRISPR/SaCas9.
Balboa D, Weltner J, Novik Y, Eurola S, Wartiovaara K, Otonkoski T.
Stem Cell Res. 2017 Jul;22:16-19. doi: 10.1016/j.scr.2017.05.005. Epub 2017 May 17.
PubMed
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| Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9. Pinto BS, Saxena T, Oliveira R, Mendez-Gomez HR, Cleary JD, Denes LT, McConnell O, Arboleda J, Xia G, Swanson MS, Wang ET. Mol Cell. 2017 Nov 2;68(3):479-490.e5. doi: 10.1016/j.molcel.2017.09.033. Epub 2017 Oct 19. PubMed |
| Genome modification of CXCR4 by Staphylococcus aureus Cas9 renders cells resistance to HIV-1 infection. Wang Q, Chen S, Xiao Q, Liu Z, Liu S, Hou P, Zhou L, Hou W, Ho W, Li C, Wu L, Guo D. Retrovirology. 2017 Nov 15;14(1):51. doi: 10.1186/s12977-017-0375-0. PubMed |
| Towards personalised allele-specific CRISPR gene editing to treat autosomal dominant disorders. Christie KA, Courtney DG, DeDionisio LA, Shern CC, De Majumdar S, Mairs LC, Nesbit MA, Moore CBT. Sci Rep. 2017 Nov 23;7(1):16174. doi: 10.1038/s41598-017-16279-4. PubMed |
| Divergent susceptibilities to AAV-SaCas9-gRNA vector-mediated genome-editing in a single-cell-derived cell population. Morsy SG, Tonne JM, Zhu Y, Lu B, Budzik K, Krempski JW, Ali SA, El-Feky MA, Ikeda Y. BMC Res Notes. 2017 Dec 8;10(1):720. doi: 10.1186/s13104-017-3028-4. PubMed |
Multimode drug inducible CRISPR/Cas9 devices for transcriptional activation and genome editing.
Lu J, Zhao C, Zhao Y, Zhang J, Zhang Y, Chen L, Han Q, Ying Y, Peng S, Ai R, Wang Y.
Nucleic Acids Res. 2018 Mar 16;46(5):e25. doi: 10.1093/nar/gkx1222.
PubMed
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| SaCas9 Requires 5'-NNGRRT-3' PAM for Sufficient Cleavage and Possesses Higher Cleavage Activity than SpCas9 or FnCpf1 in Human Cells. Xie H, Tang L, He X, Liu X, Zhou C, Liu J, Ge X, Li J, Liu C, Zhao J, Qu J, Song Z, Gu F. Biotechnol J. 2018 Apr;13(4):e1700561. doi: 10.1002/biot.201700561. Epub 2018 Jan 10. PubMed |
| In vivo genome editing improves motor function and extends survival in a mouse model of ALS. Gaj T, Ojala DS, Ekman FK, Byrne LC, Limsirichai P, Schaffer DV. Sci Adv. 2017 Dec 20;3(12):eaar3952. doi: 10.1126/sciadv.aar3952. eCollection 2017 Dec. PubMed |
| Subretinal Human Umbilical Tissue-Derived Cell Transplantation Preserves Retinal Synaptic Connectivity and Attenuates Muller Glial Reactivity. Koh S, Chen WJ, Dejneka NS, Harris IR, Lu B, Girman S, Saylor J, Wang S, Eroglu C. J Neurosci. 2018 Mar 21;38(12):2923-2943. doi: 10.1523/JNEUROSCI.1532-17.2018. Epub 2018 Feb 5. PubMed |
| Inhibition of hepatitis B virus replication via HBV DNA cleavage by Cas9 from Staphylococcus aureus. Liu Y, Zhao M, Gong M, Xu Y, Xie C, Deng H, Li X, Wu H, Wang Z. Antiviral Res. 2018 Apr;152:58-67. doi: 10.1016/j.antiviral.2018.02.011. Epub 2018 Feb 16. PubMed |
| Genome scale screening identification of SaCas9/gRNAs for targeting HIV-1 provirus and suppression of HIV-1 infection. Wang Q, Liu S, Liu Z, Ke Z, Li C, Yu X, Chen S, Guo D. Virus Res. 2018 May 2;250:21-30. doi: 10.1016/j.virusres.2018.04.002. Epub 2018 Apr 3. PubMed |
| Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy. Ryu N, Kim MA, Park D, Lee B, Kim YR, Kim KH, Baek JI, Kim WJ, Lee KY, Kim UK. Nanomedicine. 2018 Oct;14(7):2095-2102. doi: 10.1016/j.nano.2018.06.009. Epub 2018 Jun 30. PubMed |
| Thrombospondin receptor alpha2delta-1 promotes synaptogenesis and spinogenesis via postsynaptic Rac1. Risher WC, Kim N, Koh S, Choi JE, Mitev P, Spence EF, Pilaz LJ, Wang D, Feng G, Silver DL, Soderling SH, Yin HH, Eroglu C. J Cell Biol. 2018 Oct 1;217(10):3747-3765. doi: 10.1083/jcb.201802057. Epub 2018 Jul 27. PubMed |
| Ecel1 Knockdown With an AAV2-Mediated CRISPR/Cas9 System Promotes Optic Nerve Damage-Induced RGC Death in the Mouse Retina. Sato K, Shiga Y, Nakagawa Y, Fujita K, Nishiguchi KM, Tawarayama H, Murayama N, Maekawa S, Yabana T, Omodaka K, Katayama S, Feng Q, Tsuda S, Nakazawa T. Invest Ophthalmol Vis Sci. 2018 Aug 1;59(10):3943-3951. doi: 10.1167/iovs.18-23784. PubMed |
| Disruption by SaCas9 Endonuclease of HERV-Kenv, a Retroviral Gene with Oncogenic and Neuropathogenic Potential, Inhibits Molecules Involved in Cancer and Amyotrophic Lateral Sclerosis. Ibba G, Piu C, Uleri E, Serra C, Dolei A. Viruses. 2018 Aug 7;10(8). pii: v10080412. doi: 10.3390/v10080412. PubMed |
| CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo. Duchene BL, Cherif K, Iyombe-Engembe JP, Guyon A, Rousseau J, Ouellet DL, Barbeau X, Lague P, Tremblay JP. Mol Ther. 2018 Nov 7;26(11):2604-2616. doi: 10.1016/j.ymthe.2018.08.010. Epub 2018 Aug 16. PubMed |
| Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9. Wang Y, Hao L, Wang H, Santostefano K, Thapa A, Cleary J, Li H, Guo X, Terada N, Ashizawa T, Xia G. Mol Ther. 2018 Nov 7;26(11):2617-2630. doi: 10.1016/j.ymthe.2018.09.003. Epub 2018 Sep 11. PubMed |
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice.
Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Haberle J, Matos J, Robinson MD, Thony B, Schwank G.
Nat Med. 2018 Oct;24(10):1519-1525. doi: 10.1038/s41591-018-0209-1. Epub 2018 Oct 8.
PubMed
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HIT-Cas9: A CRISPR/Cas9 Genome-Editing Device under Tight and Effective Drug Control.
Zhao C, Zhao Y, Zhang J, Lu J, Chen L, Zhang Y, Ying Y, Xu J, Wei S, Wang Y.
Mol Ther Nucleic Acids. 2018 Dec 7;13:208-219. doi: 10.1016/j.omtn.2018.08.022. Epub 2018 Sep 1.
PubMed
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Orthogonal Cas9-Cas9 chimeras provide a versatile platform for genome editing.
Bolukbasi MF, Liu P, Luk K, Kwok SF, Gupta A, Amrani N, Sontheimer EJ, Zhu LJ, Wolfe SA.
Nat Commun. 2018 Nov 19;9(1):4856. doi: 10.1038/s41467-018-07310-x.
PubMed
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Creation of versatile cloning platforms for transgene expression and dCas9-based epigenome editing.
Haldeman JM, Conway AE, Arlotto ME, Slentz DH, Muoio DM, Becker TC, Newgard CB.
Nucleic Acids Res. 2018 Dec 27. pii: 5264291. doi: 10.1093/nar/gky1286.
PubMed
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A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing.
Li A, Lee CM, Hurley AE, Jarrett KE, De Giorgi M, Lu W, Balderrama KS, Doerfler AM, Deshmukh H, Ray A, Bao G, Lagor WR.
Mol Ther Methods Clin Dev. 2018 Dec 6;12:111-122. doi: 10.1016/j.omtm.2018.11.009. eCollection 2019 Mar 15.
PubMed
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| Combining orthogonal CRISPR and CRISPRi systems for genome engineering and metabolic pathway modulation in Escherichia coli. Sung LY, Wu MY, Lin MW, Hsu MN, Truong VA, Shen CC, Tu Y, Hwang KY, Tu AP, Chang YH, Hu YC. Biotechnol Bioeng. 2019 May;116(5):1066-1079. doi: 10.1002/bit.26915. Epub 2019 Feb 8. PubMed |
| The RNA-Binding Protein PUM2 Impairs Mitochondrial Dynamics and Mitophagy During Aging. D'Amico D, Mottis A, Potenza F, Sorrentino V, Li H, Romani M, Lemos V, Schoonjans K, Zamboni N, Knott G, Schneider BL, Auwerx J. Mol Cell. 2019 Feb 21;73(4):775-787.e10. doi: 10.1016/j.molcel.2018.11.034. Epub 2019 Jan 11. PubMed |
Delivering SaCas9 mRNA by lentivirus-like bionanoparticles for transient expression and efficient genome editing.
Lu B, Javidi-Parsijani P, Makani V, Mehraein-Ghomi F, Sarhan WM, Sun D, Yoo KW, Atala ZP, Lyu P, Atala A.
Nucleic Acids Res. 2019 Feb 13. pii: 5316732. doi: 10.1093/nar/gkz093.
PubMed
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| CRISPR/Cas9-mediated genome editing of splicing mutation causing congenital hearing loss. Ryu N, Kim MA, Choi DG, Kim YR, Sonn JK, Lee KY, Kim UK. Gene. 2019 Jun 30;703:83-90. doi: 10.1016/j.gene.2019.03.020. Epub 2019 Mar 18. PubMed |
| Rapid and Simple Screening of CRISPR Guide RNAs (gRNAs) in Cultured Cells Using Adeno-Associated Viral (AAV) Vectors. Fakhiri J, Nickl M, Grimm D. Methods Mol Biol. 2019;1961:111-126. doi: 10.1007/978-1-4939-9170-9_8. PubMed |
| Efficient Human Genome Editing Using SaCas9 Ribonucleoprotein Complexes. Wang Y, Wang B, Xie H, Ren Q, Liu X, Li F, Lv X, He X, Cheng C, Deng R, Li J, Zhao J, Song Z, Gu F. Biotechnol J. 2019 Jul;14(7):e1800689. doi: 10.1002/biot.201800689. Epub 2019 May 17. PubMed |
| Optimizing genome editing strategy by primer-extension-mediated sequencing. Yin J, Liu M, Liu Y, Wu J, Gan T, Zhang W, Li Y, Zhou Y, Hu J. Cell Discov. 2019 Mar 26;5:18. doi: 10.1038/s41421-019-0088-8. eCollection 2019. PubMed |
| Ex Vivo Hepatocyte Reprograming Promotes Homology-Directed DNA Repair to Correct Metabolic Disease in Mice After Transplantation. VanLith CJ, Guthman RM, Nicolas CT, Allen KL, Liu Y, Chilton JA, Tritz ZP, Nyberg SL, Kaiser RA, Lillegard JB, Hickey RD. Hepatol Commun. 2019 Feb 15;3(4):558-573. doi: 10.1002/hep4.1315. eCollection 2019 Apr. PubMed |
| CRISPR-Cas9-Mediated Correction of the 1.02 kb Common Deletion in CLN3 in Induced Pluripotent Stem Cells from Patients with Batten Disease. Burnight ER, Bohrer LR, Giacalone JC, Klaahsen DL, Daggett HT, East JS, Madumba RA, Worthington KS, Mullins RF, Stone EM, Tucker BA, Wiley LA. CRISPR J. 2018 Feb;1:75-87. doi: 10.1089/crispr.2017.0015. PubMed |
| Genome Editing in Mammalian Cell Lines using CRISPR-Cas. Liu KI, Sutrisnoh NB, Wang Y, Tan MH. J Vis Exp. 2019 Apr 11;(146). doi: 10.3791/59086. PubMed |
Targeted Transgene Activation in the Brain Tissue by Systemic Delivery of Engineered AAV1 Expressing CRISPRa.
Lau CH, Ho JW, Lo PK, Tin C.
Mol Ther Nucleic Acids. 2019 Jun 7;16:637-649. doi: 10.1016/j.omtn.2019.04.015. Epub 2019 Apr 23.
PubMed
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| Global transcriptomic analysis of the arcuate nucleus following chronic glucocorticoid treatment. Wray JR, Davies A, Sefton C, Allen TJ, Adamson A, Chapman P, Lam BYH, Yeo GSH, Coll AP, Harno E, White A. Mol Metab. 2019 Aug;26:5-17. doi: 10.1016/j.molmet.2019.05.008. Epub 2019 May 18. PubMed |
| CCR5 editing by Staphylococcus aureus Cas9 in human primary CD4(+) T cells and hematopoietic stem/progenitor cells promotes HIV-1 resistance and CD4(+) T cell enrichment in humanized mice. Xiao Q, Chen S, Wang Q, Liu Z, Liu S, Deng H, Hou W, Wu D, Xiong Y, Li J, Guo D. Retrovirology. 2019 Jun 11;16(1):15. doi: 10.1186/s12977-019-0477-y. PubMed |
Programmable RNA-Guided RNA Effector Proteins Built from Human Parts.
Rauch S, He E, Srienc M, Zhou H, Zhang Z, Dickinson BC.
Cell. 2019 Jun 27;178(1):122-134.e12. doi: 10.1016/j.cell.2019.05.049. Epub 2019 Jun 20.
PubMed
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CRISPR-READI: Efficient Generation of Knockin Mice by CRISPR RNP Electroporation and AAV Donor Infection.
Chen S, Sun S, Moonen D, Lee C, Lee AY, Schaffer DV, He L.
Cell Rep. 2019 Jun 25;27(13):3780-3789.e4. doi: 10.1016/j.celrep.2019.05.103.
PubMed
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| Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice. Lo Scrudato M, Poulard K, Sourd C, Tome S, Klein AF, Corre G, Huguet A, Furling D, Gourdon G, Buj-Bello A. Mol Ther. 2019 Aug 7;27(8):1372-1388. doi: 10.1016/j.ymthe.2019.05.021. Epub 2019 Jun 5. PubMed |
| Sequential LASER ART and CRISPR Treatments Eliminate HIV-1 in a Subset of Infected Humanized Mice. Dash PK, Kaminski R, Bella R, Su H, Mathews S, Ahooyi TM, Chen C, Mancuso P, Sariyer R, Ferrante P, Donadoni M, Robinson JA, Sillman B, Lin Z, Hilaire JR, Banoub M, Elango M, Gautam N, Mosley RL, Poluektova LY, McMillan J, Bade AN, Gorantla S, Sariyer IK, Burdo TH, Young WB, Amini S, Gordon J, Jacobson JM, Edagwa B, Khalili K, Gendelman HE. Nat Commun. 2019 Jul 2;10(1):2753. doi: 10.1038/s41467-019-10366-y. PubMed |
Delivering Cas9/sgRNA ribonucleoprotein (RNP) by lentiviral capsid-based bionanoparticles for efficient 'hit-and-run' genome editing.
Lyu P, Javidi-Parsijani P, Atala A, Lu B.
Nucleic Acids Res. 2019 Jul 12. pii: 5531787. doi: 10.1093/nar/gkz605.
PubMed
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| Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy. Moreno AM, Palmer N, Aleman F, Chen G, Pla A, Jiang N, Leong Chew W, Law M, Mali P. Nat Biomed Eng. 2019 Oct;3(10):806-816. doi: 10.1038/s41551-019-0431-2. Epub 2019 Jul 22. PubMed |
Single-Strand Annealing Plays a Major Role in Double-Strand DNA Break Repair following CRISPR-Cas9 Cleavage in Leishmania.
Zhang WW, Matlashewski G.
mSphere. 2019 Aug 21;4(4). pii: 4/4/e00408-19. doi: 10.1128/mSphere.00408-19.
PubMed
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| CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model. Ekman FK, Ojala DS, Adil MM, Lopez PA, Schaffer DV, Gaj T. Mol Ther Nucleic Acids. 2019 Sep 6;17:829-839. doi: 10.1016/j.omtn.2019.07.009. Epub 2019 Jul 26. PubMed |
| High levels of AAV vector integration into CRISPR-induced DNA breaks. Hanlon KS, Kleinstiver BP, Garcia SP, Zaborowski MP, Volak A, Spirig SE, Muller A, Sousa AA, Tsai SQ, Bengtsson NE, Loov C, Ingelsson M, Chamberlain JS, Corey DP, Aryee MJ, Joung JK, Breakefield XO, Maguire CA, Gyorgy B. Nat Commun. 2019 Sep 30;10(1):4439. doi: 10.1038/s41467-019-12449-2. PubMed |
| Efficient cleavage resolves PAM preferences of CRISPR-Cas in human cells. Tang L, Yang F, He X, Xie H, Liu X, Fu J, Xi H, Lu X, Liu C, Song Z, Qu J, Zhao J, Gu F. Cell Regen (Lond). 2019 Oct 28;8(2):44-50. doi: 10.1016/j.cr.2019.08.002. eCollection 2019 Dec. PubMed |
Anti-CRISPR AcrIIA5 Potently Inhibits All Cas9 Homologs Used for Genome Editing.
Garcia B, Lee J, Edraki A, Hidalgo-Reyes Y, Erwood S, Mir A, Trost CN, Seroussi U, Stanley SY, Cohn RD, Claycomb JM, Sontheimer EJ, Maxwell KL, Davidson AR.
Cell Rep. 2019 Nov 12;29(7):1739-1746.e5. doi: 10.1016/j.celrep.2019.10.017.
PubMed
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| Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids. Huang Q, Chan KY, Tobey IG, Chan YA, Poterba T, Boutros CL, Balazs AB, Daneman R, Bloom JM, Seed C, Deverman BE. PLoS One. 2019 Nov 14;14(11):e0225206. doi: 10.1371/journal.pone.0225206. eCollection 2019. PubMed |
| Herpesviral lytic gene functions render the viral genome susceptible to novel editing by CRISPR/Cas9. Oh HS, Neuhausser WM, Eggan P, Angelova M, Kirchner R, Eggan KC, Knipe DM. Elife. 2019 Dec 2;8. pii: 51662. doi: 10.7554/eLife.51662. PubMed |
| Haplotyping by CRISPR-mediated DNA circularization (CRISPR-hapC) broadens allele-specific gene editing. Yu J, Xiang X, Huang J, Liang X, Pan X, Dong Z, Petersen TS, Qu K, Yang L, Zhao X, Li S, Zheng T, Xu Z, Liu C, Han P, Xu F, Yang H, Liu X, Zhang X, Bolund L, Luo Y, Lin L. Nucleic Acids Res. 2020 Mar 18;48(5):e25. doi: 10.1093/nar/gkz1233. PubMed |
| Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice. Nishiguchi KM, Fujita K, Miya F, Katayama S, Nakazawa T. Nat Commun. 2020 Jan 24;11(1):482. doi: 10.1038/s41467-019-14181-3. PubMed |
| Factors Impacting Efficacy of AAV-Mediated CRISPR-Based Genome Editing for Treatment of Choroidal Neovascularization. Chung SH, Mollhoff IN, Nguyen U, Nguyen A, Stucka N, Tieu E, Manna S, Meleppat RK, Zhang P, Nguyen EL, Fong J, Zawadzki R, Yiu G. Mol Ther Methods Clin Dev. 2020 Jan 23;17:409-417. doi: 10.1016/j.omtm.2020.01.006. eCollection 2020 Jun 12. PubMed |
| Estrogen receptor-alpha expressing neurons in the ventrolateral VMH regulate glucose balance. He Y, Xu P, Wang C, Xia Y, Yu M, Yang Y, Yu K, Cai X, Qu N, Saito K, Wang J, Hyseni I, Robertson M, Piyarathna B, Gao M, Khan SA, Liu F, Chen R, Coarfa C, Zhao Z, Tong Q, Sun Z, Xu Y. Nat Commun. 2020 May 1;11(1):2165. doi: 10.1038/s41467-020-15982-7. PubMed |
Repurposing type I-F CRISPR-Cas system as a transcriptional activation tool in human cells.
Chen Y, Liu J, Zhi S, Zheng Q, Ma W, Huang J, Liu Y, Liu D, Liang P, Songyang Z.
Nat Commun. 2020 Jun 19;11(1):3136. doi: 10.1038/s41467-020-16880-8.
PubMed
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| Live-imaging of revertant and therapeutically restored dystrophin in the Dmd(EGFP-mdx) mouse model for Duchenne muscular dystrophy. Petkova MV, Stantzou A, Morin A, Petrova O, Morales-Gonzalez S, Seifert F, Bellec-Dyevre J, Manoliu T, Goyenvalle A, Garcia L, Richard I, Laplace-Builhe C, Schuelke M, Amthor H. Neuropathol Appl Neurobiol. 2020 Jun 23. doi: 10.1111/nan.12639. PubMed |
| High-fidelity SaCas9 identified by directional screening in human cells. Xie H, Ge X, Yang F, Wang B, Li S, Duan J, Lv X, Cheng C, Song Z, Liu C, Zhao J, Zhang Y, Wu J, Gao C, Zhang J, Gu F. PLoS Biol. 2020 Jul 9;18(7):e3000747. doi: 10.1371/journal.pbio.3000747. eCollection 2020 Jul. PubMed |
| Promoter Orientation within an AAV-CRISPR Vector Affects Cas9 Expression and Gene Editing Efficiency. Fry LE, Peddle CF, Stevanovic M, Barnard AR, McClements ME, MacLaren RE. CRISPR J. 2020 Aug;3(4):276-283. doi: 10.1089/crispr.2020.0021. PubMed |
| Comparison of CRISPR/Cas Endonucleases for in vivo Retinal Gene Editing. Li F, Wing K, Wang JH, Luu CD, Bender JA, Chen J, Wang Q, Lu Q, Nguyen Tran MT, Young KM, Wong RCB, Pebay A, Cook AL, Hung SSC, Liu GS, Hewitt AW. Front Cell Neurosci. 2020 Sep 10;14:570917. doi: 10.3389/fncel.2020.570917. eCollection 2020. PubMed |
| CRISPR/Cas9 as an antiviral against Orthopoxviruses using an AAV vector. Siegrist CM, Kinahan SM, Settecerri T, Greene AC, Santarpia JL. Sci Rep. 2020 Nov 9;10(1):19307. doi: 10.1038/s41598-020-76449-9. PubMed |
| Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells. Sengupta K, Mishra MK, Loro E, Spencer MJ, Pyle AD, Khurana TS. Mol Ther Nucleic Acids. 2020 Aug 29;22:500-509. doi: 10.1016/j.omtn.2020.08.031. eCollection 2020 Dec 4. PubMed |
| CRISPR based editing of SIV proviral DNA in ART treated non-human primates. Mancuso P, Chen C, Kaminski R, Gordon J, Liao S, Robinson JA, Smith MD, Liu H, Sariyer IK, Sariyer R, Peterson TA, Donadoni M, Williams JB, Siddiqui S, Bunnell BA, Ling B, MacLean AG, Burdo TH, Khalili K. Nat Commun. 2020 Nov 27;11(1):6065. doi: 10.1038/s41467-020-19821-7. PubMed |
| CRISPR/Cas9-mediated mutagenesis at microhomologous regions of human mitochondrial genome. Wang B, Lv X, Wang Y, Wang Z, Liu Q, Lu B, Liu Y, Gu F. Sci China Life Sci. 2021 Jan 6. pii: 10.1007/s11427-020-1819-8. doi: 10.1007/s11427-020-1819-8. PubMed |
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| A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation. Zhang Y, Nishiyama T, Li H, Huang J, Atmanli A, Sanchez-Ortiz E, Wang Z, Mireault AA, Mammen PPA, Bassel-Duby R, Olson EN. Mol Ther Methods Clin Dev. 2021 Jun 4;22:122-132. doi: 10.1016/j.omtm.2021.05.014. eCollection 2021 Sep 10. PubMed |
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| Antiviral Targeting of Varicella Zoster Virus Replication and Neuronal Reactivation Using CRISPR/Cas9 Cleavage of the Duplicated Open Reading Frames 62/71. Wu BW, Yee MB, Goldstein RS, Kinchington PR. Viruses. 2022 Feb 12;14(2). pii: v14020378. doi: 10.3390/v14020378. PubMed |
| Precision targeting tumor cells using cancer-specific InDel mutations with CRISPR-Cas9. Kwon T, Ra JS, Lee S, Baek IJ, Khim KW, Lee EA, Song EK, Otarbayev D, Jung W, Park YH, Wie M, Bae J, Cheng H, Park JH, Kim N, Seo Y, Yun S, Kim HE, Moon HE, Paek SH, Park TJ, Park YU, Rhee H, Choi JH, Cho SW, Myung K. Proc Natl Acad Sci U S A. 2022 Mar 1;119(9). pii: 2103532119. doi: 10.1073/pnas.2103532119. PubMed |
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| Host F-Box Protein 22 Enhances the Uptake of Brucella by Macrophages and Drives a Sustained Release of Proinflammatory Cytokines through Degradation of the Anti-Inflammatory Effector Proteins of Brucella. Mazumdar V, Joshi K, Nandi BR, Namani S, Gupta VK, Radhakrishnan G. Infect Immun. 2022 May 19;90(5):e0006022. doi: 10.1128/iai.00060-22. Epub 2022 Apr 14. PubMed |
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| Deficiency in endocannabinoid synthase DAGLB contributes to early onset Parkinsonism and murine nigral dopaminergic neuron dysfunction. Liu Z, Yang N, Dong J, Tian W, Chang L, Ma J, Guo J, Tan J, Dong A, He K, Zhou J, Cinar R, Wu J, Salinas AG, Sun L, Kumar M, Sullivan BT, Oldham BB, Pitz V, Makarious MB, Ding J, Kung J, Xie C, Hawes SL, Wang L, Wang T, Chan P, Zhang Z, Le W, Chen S, Lovinger DM, Blauwendraat C, Singleton AB, Cui G, Li Y, Cai H, Tang B. Nat Commun. 2022 Jun 17;13(1):3490. doi: 10.1038/s41467-022-31168-9. PubMed |
| Expanding the CRISPR/Cas genome-editing scope in Xenopus tropicalis. Shi Z, Jiang H, Liu G, Shi S, Zhang X, Chen Y. Cell Biosci. 2022 Jul 8;12(1):104. doi: 10.1186/s13578-022-00841-3. PubMed |
Cytosine base editing systems with minimized off-target effect and molecular size.
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| Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response. He X, Zhang Z, Xue J, Wang Y, Zhang S, Wei J, Zhang C, Wang J, Urip BA, Ngan CC, Sun J, Li Y, Lu Z, Zhao H, Pei D, Li CK, Feng B. Nat Commun. 2022 Nov 25;13(1):7275. doi: 10.1038/s41467-022-34898-y. PubMed |
| Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa. Xi Z, Vats A, Sahel JA, Chen Y, Byrne LC. Nat Commun. 2022 Dec 13;13(1):7695. doi: 10.1038/s41467-022-35361-8. PubMed |
| Hypothalamic Grb10 enhances leptin signalling and promotes weight loss. Liu H, He Y, Bai J, Zhang C, Zhang F, Yang Y, Luo H, Yu M, Liu H, Tu L, Zhang N, Yin N, Han J, Yan Z, Scarcelli NA, Conde KM, Wang M, Bean JC, Potts CHS, Wang C, Hu F, Liu F, Xu Y. Nat Metab. 2023 Jan;5(1):147-164. doi: 10.1038/s42255-022-00701-x. Epub 2023 Jan 2. PubMed |
| Genome Editing of Murine Liver Hepatocytes by AAV Vector-Mediated Expression of Cas9 In Vivo. Kashiwakura Y, Ohmori T. Methods Mol Biol. 2023;2637:195-211. doi: 10.1007/978-1-0716-3016-7_15. PubMed |
| Intra-pituitary follicle-stimulating hormone signaling regulates hepatic lipid metabolism in mice. Qiao S, Alasmi S, Wyatt A, Wartenberg P, Wang H, Candlish M, Das D, Aoki M, Grunewald R, Zhou Z, Tian Q, Yu Q, Gotz V, Belkacemi A, Raza A, Ectors F, Kattler K, Gasparoni G, Walter J, Lipp P, Mollard P, Bernard DJ, Karatayli E, Karatayli SC, Lammert F, Boehm U. Nat Commun. 2023 Feb 25;14(1):1098. doi: 10.1038/s41467-023-36681-z. PubMed |
| CRISPR-assisted transcription activation by phase separation proteins. Liu J, Chen Y, Nong B, Luo X, Cui K, Tan W, Yang Y, Ma W, Liang P, Songyang Z. Protein Cell. 2023 Mar 11:pwad013. doi: 10.1093/procel/pwad013. PubMed |
| UBE3A and transsynaptic complex NRXN1-CBLN1-GluD1 in a hypothalamic VMHvl-arcuate feedback circuit regulates aggression. Nong Y, Stoppel DC, Johnson MA, Boillot M, Todorovic J, Shen J, Zhou X, Nadler MJS, Rodriguez C, Huo Y, Nagakura I, Kasper EM, Anderson MP. bioRxiv. 2023 Mar 1:2023.02.28.530462. doi: 10.1101/2023.02.28.530462. Preprint. PubMed |
| Correction of F8 intron 1 inversion in hemophilia A patient-specific iPSCs by CRISPR/Cas9 mediated gene editing. Hu Z, Wu Y, Xiao R, Zhao J, Chen Y, Wu L, Zhou M, Liang D. Front Genet. 2023 Mar 9;14:1115831. doi: 10.3389/fgene.2023.1115831. eCollection 2023. PubMed |
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| Editing efficiencies with Cas9 orthologs, Cas12a endonucleases, and temperature in rice. Illa-Berenguer E, LaFayette PR, Parrott WA. Front Genome Ed. 2023 Mar 17;5:1074641. doi: 10.3389/fgeed.2023.1074641. eCollection 2023. PubMed |
| CRISPR editing of CCR5 and HIV-1 facilitates viral elimination in antiretroviral drug-suppressed virus-infected humanized mice. Dash PK, Chen C, Kaminski R, Su H, Mancuso P, Sillman B, Zhang C, Liao S, Sravanam S, Liu H, Waight E, Guo L, Mathews S, Sariyer R, Mosley RL, Poluektova LY, Caocci M, Amini S, Gorantla S, Burdo TH, Edagwa B, Gendelman HE, Khalili K. Proc Natl Acad Sci U S A. 2023 May 9;120(19):e2217887120. doi: 10.1073/pnas.2217887120. Epub 2023 May 1. PubMed |
| Molecular basis of FAAH-OUT-associated human pain insensitivity. Mikaeili H, Habib AM, Yeung CW, Santana-Varela S, Luiz AP, Panteleeva K, Zuberi S, Athanasiou-Fragkouli A, Houlden H, Wood JN, Okorokov AL, Cox JJ. Brain. 2023 Sep 1;146(9):3851-3865. doi: 10.1093/brain/awad098. PubMed |
| Allele-specific gene-editing approach for vision loss restoration in RHO-associated retinitis pigmentosa. Liu X, Qiao J, Jia R, Zhang F, Meng X, Li Y, Yang L. Elife. 2023 Jun 5;12:e84065. doi: 10.7554/eLife.84065. PubMed |
| Strategic self-limiting production of infectious HIV particles by CRISPR in permissive cells. Liu H, Chen C, Liao S, Sohaii DK, Cruz CRY, Burdo TH, Cradick TJ, Mehta A, Barrero C, Florez M, Gordon J, Grauzam S, Dressman J, Amini S, Bollard CM, Kaminski R, Khalili K. Mol Ther Nucleic Acids. 2023 May 19;32:1010-1025. doi: 10.1016/j.omtn.2023.04.027. eCollection 2023 Jun 13. PubMed |
| CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein. Egorova TV, Polikarpova AV, Vassilieva SG, Dzhenkova MA, Savchenko IM, Velyaev OA, Shmidt AA, Soldatov VO, Pokrovskii MV, Deykin AV, Bardina MV. Mol Ther Methods Clin Dev. 2023 Jun 17;30:161-180. doi: 10.1016/j.omtm.2023.06.006. eCollection 2023 Sep 14. PubMed |
Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier.
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| Inducible nonhuman primate models of retinal degeneration for testing end-stage therapies. Ail D, Nava D, Hwang IP, Brazhnikova E, Nouvel-Jaillard C, Dentel A, Joffrois C, Rousseau L, Degardin J, Bertin S, Sahel JA, Goureau O, Picaud S, Dalkara D. Sci Adv. 2023 Aug 2;9(31):eadg8163. doi: 10.1126/sciadv.adg8163. Epub 2023 Aug 2. PubMed |
| Prevention of early-onset cardiomyopathy in Dmd exon 52-54 deletion mice by CRISPR-Cas9-mediated exon skipping. Rok M, Wong TWY, Maino E, Ahmed A, Yang G, Hyatt E, Lindsay K, Fatehi S, Marks R, Delgado-Olguin P, Ivakine EA, Cohn RD. Mol Ther Methods Clin Dev. 2023 Jul 17;30:246-258. doi: 10.1016/j.omtm.2023.07.004. eCollection 2023 Sep 14. PubMed |
| Targeted Delivery of Chimeric Antigen Receptor into T Cells via CRISPR-Mediated Homology-Directed Repair with a Dual-AAV6 Transduction System. Moco PD, Farnos O, Sharon D, Kamen AA. Curr Issues Mol Biol. 2023 Sep 22;45(10):7705-7720. doi: 10.3390/cimb45100486. PubMed |
| Endothelial YAP Mediates Hyperglycemia-Induced Platelet Hyperactivity and Arterial Thrombosis. Li Z, Zhang J, Ma Z, Zhao G, He X, Yu X, Fu Q, Wu N, Ding Z, Sun H, Zhang X, Zhu Y, Chen L, He J. Arterioscler Thromb Vasc Biol. 2024 Jan;44(1):254-270. doi: 10.1161/ATVBAHA.123.319835. Epub 2023 Nov 2. PubMed |
| Unexpected extra exon skipping in the DYSF gene during restoring the reading frame by CRISPR/Cas9. Levchenko O, Panchuk I, Kochergin-Nikitsky K, Petrova I, Nagieva S, Pilkin M, Yakovlev I, Smirnikhina S, Deev R, Lavrov A. Biosystems. 2024 Jan;235:105072. doi: 10.1016/j.biosystems.2023.105072. Epub 2023 Nov 7. PubMed |
| Myospreader improves gene editing in skeletal muscle by myonuclear propagation. Poukalov KK, Valero MC, Muscato DR, Adams LM, Chun H, Lee YI, Andrade NS, Zeier Z, Sweeney HL, Wang ET. bioRxiv [Preprint]. 2023 Nov 6:2023.11.06.565807. doi: 10.1101/2023.11.06.565807. PubMed |
| AAV-mediated editing of PMP22 rescues Charcot-Marie-Tooth disease type 1A features in patient-derived iPS Schwann cells. Yoshioka Y, Taniguchi JB, Homma H, Tamura T, Fujita K, Inotsume M, Tagawa K, Misawa K, Matsumoto N, Nakagawa M, Inoue H, Tanaka H, Okazawa H. Commun Med (Lond). 2023 Nov 28;3(1):170. doi: 10.1038/s43856-023-00400-y. PubMed |
| In vivo genome editing via CRISPR/Cas9-mediated homology-independent targeted integration for Bietti crystalline corneoretinal dystrophy treatment. Meng X, Jia R, Zhao X, Zhang F, Chen S, Yu S, Liu X, Dou H, Feng X, Zhang J, Wang N, Xu B, Yang L. Nat Commun. 2024 May 6;15(1):3773. doi: 10.1038/s41467-024-48092-9. PubMed |
| Optimization of AAV vectors for transactivator-regulated enhanced gene expression within targeted neuronal populations. Kojima L, Seiriki K, Rokujo H, Nakazawa T, Kasai A, Hashimoto H. iScience. 2024 May 3;27(6):109878. doi: 10.1016/j.isci.2024.109878. eCollection 2024 Jun 21. PubMed |
| Conditional knockout of Shank3 in the ventral CA1 by quantitative in vivo genome-editing impairs social memory in mice. Chung M, Imanaka K, Huang Z, Watarai A, Wang MY, Tao K, Ejima H, Aida T, Feng G, Okuyama T. Nat Commun. 2024 Jun 12;15(1):4531. doi: 10.1038/s41467-024-48430-x. PubMed |
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