PX552 Citations (54)
Originally described in: In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9.Swiech L, Heidenreich M, Banerjee A, Habib N, Li Y, Trombetta J, Sur M, Zhang F Nat Biotechnol. 2014 Oct 19. doi: 10.1038/nbt.3055. PubMed Journal
Articles Citing PX552
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The CRISPR/Cas9-created MDM2 T309G enhances vitreous-induced expression of MDM2 and proliferation and survival of cells.
Duan Y, Ma G, Huang X, D'Amore PA, Zhang F, Lei H.
J Biol Chem. 2016 May 31. pii: jbc.M116.729467.
PubMed
Associated Plasmids |
AAV-Mediated CRISPR/Cas Gene Editing of Retinal Cells In Vivo.
Hung SS, Chrysostomou V, Li F, Lim JK, Wang JH, Powell JE, Tu L, Daniszewski M, Lo C, Wong RC, Crowston JG, Pebay A, King AE, Bui BV, Liu GS, Hewitt AW.
Invest Ophthalmol Vis Sci. 2016 Jun 1;57(7):3470-6. doi: 10.1167/iovs.16-19316.
PubMed
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The Development of a Viral Mediated CRISPR/Cas9 System with Doxycycline Dependent gRNA Expression for Inducible In vitro and In vivo Genome Editing.
de Solis CA, Ho A, Holehonnur R, Ploski JE.
Front Mol Neurosci. 2016 Aug 18;9:70. doi: 10.3389/fnmol.2016.00070. eCollection 2016.
PubMed
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In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration.
Suzuki K, Tsunekawa Y, Hernandez-Benitez R, Wu J, Zhu J, Kim EJ, Hatanaka F, Yamamoto M, Araoka T, Li Z, Kurita M, Hishida T, Li M, Aizawa E, Guo S, Chen S, Goebl A, Soligalla RD, Qu J, Jiang T, Fu X, Jafari M, Esteban CR, Berggren WT, Lajara J, Nunez-Delicado E, Guillen P, Campistol JM, Matsuzaki F, Liu GH, Magistretti P, Zhang K, Callaway EM, Zhang K, Belmonte JC.
Nature. 2016 Dec 1;540(7631):144-149. doi: 10.1038/nature20565. Epub 2016 Nov 16.
PubMed
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Genome editing abrogates angiogenesis in vivo. Huang X, Zhou G, Wu W, Duan Y, Ma G, Song J, Xiao R, Vandenberghe L, Zhang F, D'Amore PA, Lei H. Nat Commun. 2017 Jul 24;8(1):112. doi: 10.1038/s41467-017-00140-3. PubMed |
MANF regulates hypothalamic control of food intake and body weight. Yang S, Yang H, Chang R, Yin P, Yang Y, Yang W, Huang S, Gaertig MA, Li S, Li XJ. Nat Commun. 2017 Sep 18;8(1):579. doi: 10.1038/s41467-017-00750-x. PubMed |
Site-directed RNA repair of endogenous Mecp2 RNA in neurons. Sinnamon JR, Kim SY, Corson GM, Song Z, Nakai H, Adelman JP, Mandel G. Proc Natl Acad Sci U S A. 2017 Oct 31;114(44):E9395-E9402. doi: 10.1073/pnas.1715320114. Epub 2017 Oct 16. PubMed |
Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina. Hung SS, Li F, Wang JH, King AE, Bui BV, Liu GS, Hewitt AW. Methods Mol Biol. 2018;1715:113-133. doi: 10.1007/978-1-4939-7522-8_9. PubMed |
Mental Illnesses-Associated Fxr1 and Its Negative Regulator Gsk3beta Are Modulators of Anxiety and Glutamatergic Neurotransmission.
Khlghatyan J, Evstratova A, Chamberland S, Marakhovskaia A, Bahremand A, Toth K, Beaulieu JM.
Front Mol Neurosci. 2018 Apr 12;11:119. doi: 10.3389/fnmol.2018.00119. eCollection 2018.
PubMed
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Inhibition of PKCdelta reduces amyloid-beta levels and reverses Alzheimer disease phenotypes. Du Y, Zhao Y, Li C, Zheng Q, Tian J, Li Z, Huang TY, Zhang W, Xu H. J Exp Med. 2018 Jun 4;215(6):1665-1677. doi: 10.1084/jem.20171193. Epub 2018 May 8. PubMed |
CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease. Gyorgy B, Loov C, Zaborowski MP, Takeda S, Kleinstiver BP, Commins C, Kastanenka K, Mu D, Volak A, Giedraitis V, Lannfelt L, Maguire CA, Joung JK, Hyman BT, Breakefield XO, Ingelsson M. Mol Ther Nucleic Acids. 2018 Jun 1;11:429-440. doi: 10.1016/j.omtn.2018.03.007. Epub 2018 Mar 16. PubMed |
Nucleus of the Solitary Tract Serotonin 5-HT2C Receptors Modulate Food Intake. D'Agostino G, Lyons D, Cristiano C, Lettieri M, Olarte-Sanchez C, Burke LK, Greenwald-Yarnell M, Cansell C, Doslikova B, Georgescu T, Martinez de Morentin PB, Myers MG Jr, Rochford JJ, Heisler LK. Cell Metab. 2018 Oct 2;28(4):619-630.e5. doi: 10.1016/j.cmet.2018.07.017. Epub 2018 Aug 23. PubMed |
Nuclear PGK1 Alleviates ADP-Dependent Inhibition of CDC7 to Promote DNA Replication. Li X, Qian X, Jiang H, Xia Y, Zheng Y, Li J, Huang BJ, Fang J, Qian CN, Jiang T, Zeng YX, Lu Z. Mol Cell. 2018 Nov 15;72(4):650-660.e8. doi: 10.1016/j.molcel.2018.09.007. Epub 2018 Nov 1. PubMed |
Induction of Brain Arteriovenous Malformation Through CRISPR/Cas9-Mediated Somatic Alk1 Gene Mutations in Adult Mice. Zhu W, Saw D, Weiss M, Sun Z, Wei M, Shaligram S, Wang S, Su H. Transl Stroke Res. 2018 Dec 3. pii: 10.1007/s12975-018-0676-1. doi: 10.1007/s12975-018-0676-1. PubMed |
Vasoactive intestinal peptide controls the suprachiasmatic circadian clock network via ERK1/2 and DUSP4 signalling. Hamnett R, Crosby P, Chesham JE, Hastings MH. Nat Commun. 2019 Feb 1;10(1):542. doi: 10.1038/s41467-019-08427-3. PubMed |
Screening of CRISPR/Cas base editors to target the AMD high-risk Y402H complement factor H variant. Tran MTN, Khalid MKNM, Pebay A, Cook AL, Liang HH, Wong RCB, Craig JE, Liu GS, Hung SS, Hewitt AW. Mol Vis. 2019 Mar 16;25:174-182. eCollection 2019. PubMed |
Utility of Self-Destructing CRISPR/Cas Constructs for Targeted Gene Editing in the Retina. Li F, Hung SSC, Mohd Khalid MKN, Wang JH, Chrysostomou V, Wong VHY, Singh V, Wing K, Tu L, Bender JA, Pebay A, King AE, Cook AL, Wong RCB, Bui BV, Hewitt AW, Liu GS. Hum Gene Ther. 2019 Nov;30(11):1349-1360. doi: 10.1089/hum.2019.021. Epub 2019 Oct 25. PubMed |
Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction. Suzuki K, Yamamoto M, Hernandez-Benitez R, Li Z, Wei C, Soligalla RD, Aizawa E, Hatanaka F, Kurita M, Reddy P, Ocampo A, Hishida T, Sakurai M, Nemeth AN, Nunez Delicado E, Campistol JM, Magistretti P, Guillen P, Rodriguez Esteban C, Gong J, Yuan Y, Gu Y, Liu GH, Lopez-Otin C, Wu J, Zhang K, Izpisua Belmonte JC. Cell Res. 2019 Oct;29(10):804-819. doi: 10.1038/s41422-019-0213-0. Epub 2019 Aug 23. 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 |
Neuronal programming by microbiota regulates intestinal physiology.
Obata Y, Castano A, Boeing S, Bon-Frauches AC, Fung C, Fallesen T, de Aguero MG, Yilmaz B, Lopes R, Huseynova A, Horswell S, Maradana MR, Boesmans W, Vanden Berghe P, Murray AJ, Stockinger B, Macpherson AJ, Pachnis V.
Nature. 2020 Feb;578(7794):284-289. doi: 10.1038/s41586-020-1975-8. Epub 2020 Feb 5.
PubMed
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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 |
In vivo CRISPRa decreases seizures and rescues cognitive deficits in a rodent model of epilepsy. Colasante G, Qiu Y, Massimino L, Di Berardino C, Cornford JH, Snowball A, Weston M, Jones SP, Giannelli S, Lieb A, Schorge S, Kullmann DM, Broccoli V, Lignani G. Brain. 2020 Mar 1;143(3):891-905. doi: 10.1093/brain/awaa045. PubMed |
Truncation of mutant huntingtin in knock-in mice demonstrates exon1 huntingtin is a key pathogenic form. Yang H, Yang S, Jing L, Huang L, Chen L, Zhao X, Yang W, Pan Y, Yin P, Qin ZS, Li S, Li XJ. Nat Commun. 2020 May 22;11(1):2582. doi: 10.1038/s41467-020-16318-1. PubMed |
Loss of Hap1 selectively promotes striatal degeneration in Huntington disease mice. Liu Q, Cheng S, Yang H, Zhu L, Pan Y, Jing L, Tang B, Li S, Li XJ. Proc Natl Acad Sci U S A. 2020 Aug 18;117(33):20265-20273. doi: 10.1073/pnas.2002283117. Epub 2020 Aug 3. PubMed |
D1 receptors in the anterior cingulate cortex modulate basal mechanical sensitivity threshold and glutamatergic synaptic transmission. Darvish-Ghane S, Quintana C, Beaulieu JM, Martin LJ. Mol Brain. 2020 Sep 5;13(1):121. doi: 10.1186/s13041-020-00661-x. PubMed |
AAV-mediated in vivo genome editing in vascular endothelial cells. Wu W, Yang Y, Yao F, Dong L, Xia X, Zhang S, Lei H. Methods. 2020 Dec 9. pii: S1046-2023(20)30263-2. doi: 10.1016/j.ymeth.2020.12.001. PubMed |
The mRNA-Binding Protein RBM3 Regulates Activity Patterns and Local Synaptic Translation in Cultured Hippocampal Neurons. Sertel SM, von Elling-Tammen MS, Rizzoli SO. J Neurosci. 2021 Feb 10;41(6):1157-1173. doi: 10.1523/JNEUROSCI.0921-20.2020. Epub 2020 Dec 11. PubMed |
Enkephalin release from VIP interneurons in the hippocampal CA2/3a region mediates heterosynaptic plasticity and social memory. Leroy F, de Solis CA, Boyle LM, Bock T, Lofaro OM, Buss EW, Asok A, Kandel ER, Siegelbaum SA. Mol Psychiatry. 2022 Jun;27(6):2879-2900. doi: 10.1038/s41380-021-01124-y. Epub 2021 May 14. PubMed |
Huntingtin silencing delays onset and slows progression of Huntington's disease: a biomarker study. Liu H, Zhang C, Xu J, Jin J, Cheng L, Miao X, Wu Q, Wei Z, Liu P, Lu H, van Zijl PCM, Ross CA, Hua J, Duan W. Brain. 2021 May 27. pii: 6286941. doi: 10.1093/brain/awab190. PubMed |
Anterior thalamic dysfunction underlies cognitive deficits in a subset of neuropsychiatric disease models. Roy DS, Zhang Y, Aida T, Choi S, Chen Q, Hou Y, Lea NE, Skaggs KM, Quay JC, Liew M, Maisano H, Le V, Jones C, Xu J, Kong D, Sullivan HA, Saunders A, McCarroll SA, Wickersham IR, Feng G. Neuron. 2021 Aug 18;109(16):2590-2603.e13. doi: 10.1016/j.neuron.2021.06.005. Epub 2021 Jun 30. PubMed |
Generation of Transgenic Rat Embryonic Stem Cells Using the CRISPR/Cpf1 System for Inducible Gene Knockout. Sherstyuk VV, Zakian SM. Biochemistry (Mosc). 2021 Jul;86(7):843-851. doi: 10.1134/S0006297921070051. PubMed |
Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases.
Jang H, Jo DH, Cho CS, Shin JH, Seo JH, Yu G, Gopalappa R, Kim D, Cho SR, Kim JH, Kim HH.
Nat Biomed Eng. 2021 Aug 26. pii: 10.1038/s41551-021-00788-9. doi: 10.1038/s41551-021-00788-9.
PubMed
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PINK1 kinase dysfunction triggers neurodegeneration in the primate brain without impacting mitochondrial homeostasis. Yang W, Guo X, Tu Z, Chen X, Han R, Liu Y, Yan S, Wang Q, Wang Z, Zhao X, Zhang Y, Xiong X, Yang H, Yin P, Wan H, Chen X, Guo J, Yan XX, Liao L, Li S, Li XJ. Protein Cell. 2022 Jan;13(1):26-46. doi: 10.1007/s13238-021-00888-x. Epub 2021 Nov 20. PubMed |
TTP protects against acute liver failure by regulating CCL2 and CCL5 through m6A RNA methylation. Xiao P, Li M, Zhou M, Zhao X, Wang C, Qiu J, Fang Q, Jiang H, Dong H, Zhou R. JCI Insight. 2021 Dec 8;6(23). pii: 149276. doi: 10.1172/jci.insight.149276. PubMed |
Imaging Net Retrograde Axonal Transport In Vivo: A Physiological Biomarker. Lee PJ, Kennedy Z, Wang Y, Lu Y, Cefaliello C, Uyan O, Song CQ, Godinho BMC, Xu Z, Rusckowski M, Xue W, Brown RH Jr. Ann Neurol. 2022 May;91(5):716-729. doi: 10.1002/ana.26329. Epub 2022 Mar 19. PubMed |
In vivo outer hair cell gene editing ameliorates progressive hearing loss in dominant-negative Kcnq4 murine model. Noh B, Rim JH, Gopalappa R, Lin H, Kim KM, Kang MJ, Gee HY, Choi JY, Kim HH, Jung J. Theranostics. 2022 Feb 28;12(5):2465-2482. doi: 10.7150/thno.67781. eCollection 2022. PubMed |
Knockdown of GABAA alpha3 subunits on thalamic reticular neurons enhances deep sleep in mice. Uygun DS, Yang C, Tilli ER, Katsuki F, Hodges EL, McKenna JT, McNally JM, Brown RE, Basheer R. Nat Commun. 2022 Apr 26;13(1):2246. doi: 10.1038/s41467-022-29852-x. PubMed |
CRISPR-based VEGF suppression using paired guide RNAs for treatment of choroidal neovascularization.
Chung SH, Sin TN, Dang B, Ngo T, Lo T, Lent-Schochet D, Meleppat RK, Zawadzki RJ, Yiu G.
Mol Ther Nucleic Acids. 2022 Apr 27;28:613-622. doi: 10.1016/j.omtn.2022.04.015. eCollection 2022 Jun 14.
PubMed
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Targeting thalamic circuits rescues motor and mood deficits in PD mice. Zhang Y, Roy DS, Zhu Y, Chen Y, Aida T, Hou Y, Shen C, Lea NE, Schroeder ME, Skaggs KM, Sullivan HA, Fischer KB, Callaway EM, Wickersham IR, Dai J, Li XM, Lu Z, Feng G. Nature. 2022 Jul;607(7918):321-329. doi: 10.1038/s41586-022-04806-x. Epub 2022 Jun 8. PubMed |
Neurotensin orchestrates valence assignment in the amygdala. Li H, Namburi P, Olson JM, Borio M, Lemieux ME, Beyeler A, Calhoon GG, Hitora-Imamura N, Coley AA, Libster A, Bal A, Jin X, Wang H, Jia C, Choudhury SR, Shi X, Felix-Ortiz AC, de la Fuente V, Barth VP, King HO, Izadmehr EM, Revanna JS, Batra K, Fischer KB, Keyes LR, Padilla-Coreano N, Siciliano CA, McCullough KM, Wichmann R, Ressler KJ, Fiete IR, Zhang F, Li Y, Tye KM. Nature. 2022 Aug;608(7923):586-592. doi: 10.1038/s41586-022-04964-y. Epub 2022 Jul 20. PubMed |
Allele-specific silencing of the gain-of-function mutation in Huntington's disease using CRISPR/Cas9. Shin JW, Hong EP, Park SS, Choi DE, Seong IS, Whittaker MN, Kleinstiver BP, Chen RZ, Lee JM. JCI Insight. 2022 Oct 10;7(19):e141042. doi: 10.1172/jci.insight.141042. PubMed |
PAM-altering SNP-based allele-specific CRISPR-Cas9 therapeutic strategies for Huntington's disease. Shin JW, Hong EP, Park SS, Choi DE, Zeng S, Chen RZ, Lee JM. Mol Ther Methods Clin Dev. 2022 Aug 14;26:547-561. doi: 10.1016/j.omtm.2022.08.005. eCollection 2022 Sep 8. PubMed |
G-quadruplexes sense natural porphyrin metabolites for regulation of gene transcription and chromatin landscapes. Li C, Yin Z, Xiao R, Huang B, Cui Y, Wang H, Xiang Y, Wang L, Lei L, Ye J, Li T, Zhong Y, Guo F, Xia Y, Fang P, Liang K. Genome Biol. 2022 Dec 15;23(1):259. doi: 10.1186/s13059-022-02830-8. PubMed |
Adolescent Parvalbumin Expression in the Left Orbitofrontal Cortex Shapes Sociability in Female Mice. Jeon YS, Jeong D, Kweon H, Kim JH, Kim CY, Oh Y, Lee YH, Kim CH, Kim SG, Jeong JW, Kim E, Lee SH. J Neurosci. 2023 Mar 1;43(9):1555-1571. doi: 10.1523/JNEUROSCI.0918-22.2023. Epub 2023 Jan 30. PubMed |
Inducible CRISPR Epigenome Systems Mimic Cocaine Induced Bidirectional Regulation of Nab2 and Egr3. Choi EY, Franco D, Stapf CA, Gordin M, Chow A, Cover KK, Chandra R, Lobo MK. J Neurosci. 2023 Mar 29;43(13):2242-2259. doi: 10.1523/JNEUROSCI.1802-22.2022. Epub 2023 Feb 27. PubMed |
Antibody-directed extracellular proximity biotinylation reveals Contactin-1 regulates axo-axonic innervation of axon initial segments. Ogawa Y, Lim BC, George S, Oses-Prieto JA, Rasband JM, Eshed-Eisenbach Y, Nair S, Boato F, Peles E, Burlingame AL, Aelst LV, Rasband MN. bioRxiv. 2023 Mar 6:2023.03.06.531378. doi: 10.1101/2023.03.06.531378. Preprint. PubMed |
Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease. Choi DE, Shin JW, Zeng S, Hong EP, Jang JH, Loupe JM, Wheeler VC, Stutzman HE, Kleinstiver BP, Lee JM. bioRxiv. 2023 Apr 28:2023.04.28.538700. doi: 10.1101/2023.04.28.538700. Preprint. PubMed |
Elevated levels of FMRP-target MAP1B impair human and mouse neuronal development and mouse social behaviors via autophagy pathway.
Guo Y, Shen M, Dong Q, Mendez-Albelo NM, Huang SX, Sirois CL, Le J, Li M, Jarzembowski ED, Schoeller KA, Stockton ME, Horner VL, Sousa AMM, Gao Y, Levine JE, Wang D, Chang Q, Zhao X.
Nat Commun. 2023 Jun 26;14(1):3801. doi: 10.1038/s41467-023-39337-0.
PubMed
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How chromosomal translocations arise to cause cancer: Gene proximity, trans-splicing, and DNA end joining. Streb P, Kowarz E, Benz T, Reis J, Marschalek R. iScience. 2023 May 19;26(6):106900. doi: 10.1016/j.isci.2023.106900. eCollection 2023 Jun 16. PubMed |
Visualizing Arc protein dynamics and localization in the mammalian brain using AAV-mediated in situ gene labeling. Avallone M, Pardo J, Mergiya TF, Rajova J, Rasanen A, Davidsson M, Akerblom M, Quintino L, Kumar D, Bramham CR, Bjorklund T. Front Mol Neurosci. 2023 Jun 15;16:1140785. doi: 10.3389/fnmol.2023.1140785. eCollection 2023. PubMed |
Base editing of the mutated TERT promoter inhibits liver tumor growth. Zhao G, Ma Q, Yang H, Jiang H, Xu Q, Luo S, Meng Z, Liu J, Zhu L, Lin Q, Li M, Fang J, Ma L, Qiu W, Mao Z, Lu Z. Hepatology. 2023 Nov 28. doi: 10.1097/HEP.0000000000000700. PubMed |
Personalized allele-specific CRISPR-Cas9 strategies for myofibrillar myopathy 6. Shin JW, Kim KH, Lee Y, Choi DE, Lee JM. medRxiv [Preprint]. 2024 Feb 4:2024.02.03.24302252. doi: 10.1101/2024.02.03.24302252. 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 |
Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease. Choi DE, Shin JW, Zeng S, Hong EP, Jang JH, Loupe JM, Wheeler VC, Stutzman HE, Kleinstiver B, Lee JM. Elife. 2024 Jun 13;12:RP89782. doi: 10.7554/eLife.89782. PubMed |
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