PX551 Citations (37)

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

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Articles Citing PX551

Articles
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 pAAV-RSV-SpCas9 pAAV-U6-sgRNA-CMV-GFP
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 Associated Plasmids AAV-U6-sgRNA-hSyn-mCherry
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 Associated Plasmids pJEP15-AAV-H1/TO-sgRNA(Tet2)-CMV-TetR-P2A-eGFP-KASH-pA pJEP14-AAV-H1/TO-sgRNA(Empty)-CMV-TetR-P2A-eGFP-KASH-pA pJEP13-AAV-H1/TO-L-sgRNA(Tet2)-CMV-TetR-P2A-eGFP-KASH-pA pJEP12-AAV-H1/TO-L-sgRNA(Empty)-CMV-TetR-P2A-eGFP-KASH-pA pJEP11-AAV-U6/TO-sgRNA(Tet2)-CMV-TetR-P2A-eGFP-KASH-pA More ...
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 Associated Plasmids pCAG-1BPNLS-Cas9-1BPNLS pCAG-1BPNLS-Cas9-1BPNLS-2AGFP pCAGmCherry-gRNA Tubb3gRNA-mCherry pTubb3-MC More ...
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
Virus-Mediated Genome Editing via Homology-Directed Repair in Mitotic and Postmitotic Cells in Mammalian Brain. Nishiyama J, Mikuni T, Yasuda R. Neuron. 2017 Oct 18. pii: S0896-6273(17)30933-9. doi: 10.1016/j.neuron.2017.10.004. PubMed Associated Plasmids pAAV-EFS-SpCas9 pAAV-HDR-mEGFP-camk2a pAAV-HDR-mEGFP-Actin
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
In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation. Liao HK, Hatanaka F, Araoka T, Reddy P, Wu MZ, Sui Y, Yamauchi T, Sakurai M, O'Keefe DD, Nunez-Delicado E, Guillen P, Campistol JM, Wu CJ, Lu LF, Esteban CR, Izpisua Belmonte JC. Cell. 2017 Dec 14;171(7):1495-1507.e15. doi: 10.1016/j.cell.2017.10.025. Epub 2017 Dec 7. PubMed Associated Plasmids AAV-U6-dgRNA-CAG-MPH AAV-nEF-dCas9 AAV-CMVc-Cas9 AAV-tLuc AAV-tLuc-mCherry
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 Associated Plasmids pAAV hSyn GFP-Fxr1 pAAV Gsk3 sgRNA/GFP
In vivo proximity proteomics of nascent synapses reveals a novel regulator of cytoskeleton-mediated synaptic maturation. Spence EF, Dube S, Uezu A, Locke M, Soderblom EJ, Soderling SH. Nat Commun. 2019 Jan 23;10(1):386. doi: 10.1038/s41467-019-08288-w. 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
Neuron-Specific Genome Modification in the Adult Rat Brain Using CRISPR-Cas9 Transgenic Rats. Back S, Necarsulmer J, Whitaker LR, Coke LM, Koivula P, Heathward EJ, Fortuno LV, Zhang Y, Yeh CG, Baldwin HA, Spencer MD, Mejias-Aponte CA, Pickel J, Hoffman AF, Spivak CE, Lupica CR, Underhill SM, Amara SG, Domanskyi A, Anttila JE, Airavaara M, Hope BT, Hamra FK, Richie CT, Harvey BK. Neuron. 2019 Feb 8. pii: S0896-6273(19)30062-5. doi: 10.1016/j.neuron.2019.01.035. PubMed Associated Plasmids pOTTC1032 - pAAV EF1a Nuc-flox(mCherry)-EGFP pOTTC1442 - pAAV CMV-IE Nuc-iRFP-2A-iCre pOTTC1440 - pAAV CMV-IE Nuc-iRFP-2A-Flpo pOTTC1124 - pAAV MeCP2 SpCas9(D10A) pOTTC1088 - pAAV TH gRNA A+B pair EF1a EGFP More ...
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 pAAV-pMecp2-dSaCas9-VP64-pU6-sgRNA pAAV-pMecp2-dSaCas9-VP160-pU6-sgRNA pAAV-pMecp2-dSaCas9-KRAB-pU6-sgRNA pAAV-pMecp2-dSaCas9-SID4X-pU6-sgRNA pAAV-pCMV-dSaCas9-VP64-pU6-sgRNA More ...
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
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
The KDM5A/RBP2 histone demethylase represses NOTCH signaling to sustain neuroendocrine differentiation and promote small cell lung cancer tumorigenesis. Oser MG, Sabet AH, Gao W, Chakraborty AA, Schinzel AC, Jennings RB, Fonseca R, Bonal DM, Booker MA, Flaifel A, Novak JS, Christensen CL, Zhang H, Herbert ZT, Tolstorukov MY, Buss EJ, Wong KK, Bronson RT, Nguyen QD, Signoretti S, Kaelin WG Jr. Genes Dev. 2019 Dec 1;33(23-24):1718-1738. doi: 10.1101/gad.328336.119. Epub 2019 Nov 14. 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
Delta glutamate receptor conductance drives excitation of mouse dorsal raphe neurons. Gantz SC, Moussawi K, Hake HS. Elife. 2020 Apr 1;9. pii: 56054. doi: 10.7554/eLife.56054. PubMed
Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation. Panagiotopoulos AL, Karguth N, Pavlou M, Bohm S, Gasparoni G, Walter J, Graf A, Blum H, Biel M, Riedmayr LM, Becirovic E. Mol Ther Nucleic Acids. 2020 Sep 4;21:1050-1061. doi: 10.1016/j.omtn.2020.07.036. Epub 2020 Jul 31. 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
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
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
Therapeutic homology-independent targeted integration in retina and liver. Tornabene P, Ferla R, Llado-Santaeularia M, Centrulo M, Dell'Anno M, Esposito F, Marrocco E, Pone E, Minopoli R, Iodice C, Nusco E, Rossi S, Lyubenova H, Manfredi A, Di Filippo L, Iuliano A, Torella A, Piluso G, Musacchia F, Surace EM, Cacchiarelli D, Nigro V, Auricchio A. Nat Commun. 2022 Apr 12;13(1):1963. doi: 10.1038/s41467-022-29550-8. 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 Associated Plasmids px552-U6-gRNA1-CMV-eGFP px552-U6-gRNA2-CMV-eGFP px552-U6-gRNA1-U6-gRNA2-CMV-eGFP
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
De novo pyrimidine synthesis is a targetable vulnerability in IDH mutant glioma. Shi DD, Savani MR, Levitt MM, Wang AC, Endress JE, Bird CE, Buehler J, Stopka SA, Regan MS, Lin YF, Puliyappadamba VT, Gao W, Khanal J, Evans L, Lee JH, Guo L, Xiao Y, Xu M, Huang B, Jennings RB, Bonal DM, Martin-Sandoval MS, Dang T, Gattie LC, Cameron AB, Lee S, Asara JM, Kornblum HI, Mak TW, Looper RE, Nguyen QD, Signoretti S, Gradl S, Sutter A, Jeffers M, Janzer A, Lehrman MA, Zacharias LG, Mathews TP, Losman JA, Richardson TE, Cahill DP, DeBerardinis RJ, Ligon KL, Xu L, Ly P, Agar NYR, Abdullah KG, Harris IS, Kaelin WG Jr, McBrayer SK. Cancer Cell. 2022 Sep 12;40(9):939-956.e16. doi: 10.1016/j.ccell.2022.07.011. Epub 2022 Aug 18. PubMed Associated Plasmids pMSCV-MerCreMer-hygro pAAV2-sgTrp53-sgAtrx-EFS-Cre pLBCX-Large-T-antigenK1 mutant
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
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
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
Oxytocinergic neurons, but not oxytocin, are crucial for male penile erection. Wen YX, Fan LY, Yang AY, Zhang YC, Xu C, Wang ZH, Xie WJ, Lu Y, Zhang XY, Zhu JN, Sun A, Li L, Zhang QP. Neuropharmacology. 2023 Sep 1;235:109576. doi: 10.1016/j.neuropharm.2023.109576. Epub 2023 May 8. PubMed
Ceramide compensation by ceramide synthases preserves retinal function and structure in a retinal dystrophy mouse model. Qian X, Srinivasan T, He J, Lu J, Jin Y, Gu H, Chen R. Dis Model Mech. 2023 Jul 1;16(7):dmm050168. doi: 10.1242/dmm.050168. Epub 2023 Jul 19. PubMed
Acoustically targeted noninvasive gene therapy in large brain volumes. Nouraein S, Lee S, Saenz VA, Del Mundo HC, Yiu J, Szablowski JO. Gene Ther. 2023 Sep 12. doi: 10.1038/s41434-023-00421-1. 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
Intranasal delivery of shRNA to knockdown the 5HT-2A receptor enhances memory and alleviates anxiety. Rohn TT, Radin D, Brandmeyer T, Seidler PG, Linder BJ, Lytle T, Mee JL, Macciardi F. Transl Psychiatry. 2024 Mar 20;14(1):154. doi: 10.1038/s41398-024-02879-y. 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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