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pX601-AAV-CMV::NLS-SaCas9-NLS-3xHA-bGHpA;U6::BsaI-sgRNA Citations (90)

Plasmid Article: 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

Articles
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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

The Development of an AAV-Based CRISPR SaCas9 Genome Editing System That Can Be Delivered to Neurons in vivo and Regulated via Doxycycline and Cre-Recombinase. Kumar N, Stanford W, de Solis C, Aradhana, Abraham ND, Dao TJ, Thaseen S, Sairavi A, Gonzalez CU, Ploski JE. Front Mol Neurosci. 2018 Nov 13;11:413. doi: 10.3389/fnmol.2018.00413. eCollection 2018. PubMed
A Transcription Factor Addiction in Leukemia Imposed by the MLL Promoter Sequence. Lu B, Klingbeil O, Tarumoto Y, Somerville TDD, Huang YH, Wei Y, Wai DC, Low JKK, Milazzo JP, Wu XS, Cao Z, Yan X, Demerdash OE, Huang G, Mackay JP, Kinney JB, Shi J, Vakoc CR. Cancer Cell. 2018 Dec 10;34(6):970-981.e8. doi: 10.1016/j.ccell.2018.10.015. Epub 2018 Nov 29. PubMed
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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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
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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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

Associated Plasmids

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
Expanding the CRISPR Toolbox with ErCas12a in Zebrafish and Human Cells. Wierson WA, Simone BW, WareJoncas Z, Mann C, Welker JM, Kar B, Emch MJ, Friedberg I, Gendron WAC, Barry MA, Clark KJ, Dobbs DL, McGrail MA, Ekker SC, Essner JJ. CRISPR J. 2019 Dec;2(6):417-433. doi: 10.1089/crispr.2019.0026. Epub 2019 Nov 19. 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

Associated Plasmids

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
CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice. Stone D, Long KR, Loprieno MA, De Silva Feelixge HS, Kenkel EJ, Liley RM, Rapp S, Roychoudhury P, Nguyen T, Stensland L, Colon-Thillet R, Klouser LM, Weber ND, Le C, Wagoner J, Goecker EA, Li AZ, Eichholz K, Corey L, Tyrrell DL, Greninger AL, Huang ML, Polyak SJ, Aubert M, Sagartz JE, Jerome KR. Mol Ther Methods Clin Dev. 2020 Nov 26;20:258-275. doi: 10.1016/j.omtm.2020.11.014. eCollection 2021 Mar 12. PubMed
A hypomorphic variant in EYS detected by genome-wide association study contributes toward retinitis pigmentosa. Nishiguchi KM, Miya F, Mori Y, Fujita K, Akiyama M, Kamatani T, Koyanagi Y, Sato K, Takigawa T, Ueno S, Tsugita M, Kunikata H, Cisarova K, Nishino J, Murakami A, Abe T, Momozawa Y, Terasaki H, Wada Y, Sonoda KH, Rivolta C, Tsunoda T, Tsujikawa M, Ikeda Y, Nakazawa T. Commun Biol. 2021 Jan 29;4(1):140. doi: 10.1038/s42003-021-01662-9. PubMed
AAV-Mediated CRISPRi and RNAi Based Gene Silencing in Mouse Hippocampal Neurons. Deutsch M, Gunther A, Lerchundi R, Rose CR, Balfanz S, Baumann A. Cells. 2021 Feb 4;10(2). pii: cells10020324. doi: 10.3390/cells10020324. PubMed
Discovery and engineering of small SlugCas9 with broad targeting range and high specificity and activity. Hu Z, Zhang C, Wang S, Gao S, Wei J, Li M, Hou L, Mao H, Wei Y, Qi T, Liu H, Liu D, Lan F, Lu D, Wang H, Li J, Wang Y. Nucleic Acids Res. 2021 Mar 15. pii: 6171296. doi: 10.1093/nar/gkab148. PubMed

Associated Plasmids

Generation of a homozygous LRP2 knockout human embryonic stem cell line (FDCHDPe010-A-56) by CRISPR/Cas9 system. You J, Cheng Y, Yang XJ, Chen L. Stem Cell Res. 2021 May;53:102342. doi: 10.1016/j.scr.2021.102342. Epub 2021 Apr 8. PubMed
Comprehensive optimization of a reporter assay toolbox for three distinct CRISPR-Cas systems. Chen L, Gao H, Zhou B, Wang Y. FEBS Open Bio. 2021 Jul;11(7):1965-1980. doi: 10.1002/2211-5463.13198. Epub 2021 Jun 9. PubMed
In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment. Hong SA, Seo JH, Wi S, Jung ES, Yu J, Hwang GH, Yu JH, Baek A, Park S, Bae S, Cho SR. Mol Ther. 2022 Jan 5;30(1):119-129. doi: 10.1016/j.ymthe.2021.05.022. Epub 2021 May 29. PubMed
Inhibition of Hepatitis B Virus by AAV8-Derived CRISPR/SaCas9 Expressed From Liver-Specific Promoters. Yan K, Feng J, Liu X, Wang H, Li Q, Li J, Xu T, Sajid M, Ullah H, Zhou L, Zhou L, Chen Y. Front Microbiol. 2021 Jun 26;12:665184. doi: 10.3389/fmicb.2021.665184. eCollection 2021. PubMed
Dual-AAV delivering split prime editor system for in vivo genome editing. Zhi S, Chen Y, Wu G, Wen J, Wu J, Liu Q, Li Y, Kang R, Hu S, Wang J, Liang P, Huang J. Mol Ther. 2021 Jul 21. pii: S1525-0016(21)00365-8. doi: 10.1016/j.ymthe.2021.07.011. PubMed
Unlocking loxP to Track Genome Editing In Vivo. Gendron WAC, Rubin JD, Hansen MJ, Nace RA, Simone BW, Ekker SC, Barry MA. Genes (Basel). 2021 Aug 3;12(8). pii: genes12081204. doi: 10.3390/genes12081204. PubMed
Adeno-Associated Vector-Delivered CRISPR/SaCas9 System Reduces Feline Leukemia Virus Production In Vitro. Helfer-Hungerbuehler AK, Shah J, Meili T, Boenzli E, Li P, Hofmann-Lehmann R. Viruses. 2021 Aug 18;13(8). pii: v13081636. doi: 10.3390/v13081636. PubMed
CRISPR/Cas9-Mediated in vivo Genetic Correction in a Mouse Model of Hemophilia A. Luo S, Li Z, Dai X, Zhang R, Liang Z, Li W, Zeng M, Su J, Wang J, Liang X, Wu Y, Liang D. Front Cell Dev Biol. 2021 Aug 16;9:672564. doi: 10.3389/fcell.2021.672564. eCollection 2021. PubMed
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
scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution. Ozturk BE, Johnson ME, Kleyman M, Turunc S, He J, Jabalameli S, Xi Z, Visel M, Dufour VL, Iwabe S, Pompeo Marinho LFL, Aguirre GD, Sahel JA, Schaffer DV, Pfenning AR, Flannery JG, Beltran WA, Stauffer WR, Byrne LC. Elife. 2021 Oct 19;10. pii: 64175. doi: 10.7554/eLife.64175. PubMed
Compact SchCas9 Recognizes the Simple NNGR PAM. Wang S, Mao H, Hou L, Hu Z, Wang Y, Qi T, Tao C, Yang Y, Zhang C, Li M, Liu H, Hu S, Chai R, Wang Y. Adv Sci (Weinh). 2022 Feb;9(4):e2104789. doi: 10.1002/advs.202104789. Epub 2021 Dec 6. PubMed

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