...92247 pJOP-HTT-HR30Q HTT copGFP E1Fa Huntington's Mahmoud Pouladi 92248 pJOP-HTT-HR45Q HTT copGFP E1Fa...92249 pJOP-HTT-HR65Q HTT copGFP E1Fa Huntington's Mahmoud Pouladi 92250 pJOP-HTT-HR81Q HTT copGFP E1Fa...His6-Ssp-Httex1-7Q HTT His T7 Huntington's Hilal Lashuel 84348 pTWIN1-His6-Ssp-Httex1-15Q HTT His T7 Huntington's...His6-Ssp-Httex1-23Q HTT His T7 Huntington's Hilal Lashuel 84350 pTWIN1-His6-Ssp-Httex1-29Q HTT His T7 Huntington's...His6-Ssp-Httex1-37Q HTT His T7 Huntington's Hilal Lashuel 84352 pTWIN1-His6-Ssp-Httex1-43Q HTT His T7 Huntington's...Garcia 157860 61427-sgHtt_6 HTT U6 Huntington's Xinyu Zhao 157861 61427-sgHtt_10 HTT U6 Huntington's Xinyu...-U6-sgHTT1-7sk-sgCas9 HTT U6, 7sk Huntington's Nicole Deglon 190901 pAAVss-U6-sgHTT51-7sk-Cas9 HTT U6,...

...series within the huntingtin (HTT) gene. An individual with the mutated HTT gene will have huntingtin proteins...neurodegenerative disorders. Find plasmids expressing HTT, C9ORF72, LRRK2, Synuclein, and more. New and Noteworthy...

..., AmpR Related Resources SGC: http://www.thesgc.org/ SGC Toronto: https://www.thesgc.org/scientists/groups...groups/toronto SGC Oxford: http://www.sgc.ox.ac.uk/ Protein Database (PDB): http://www.pdb.org Request Plasmids...

...hundred kilobases. Nature Methods , 6(5), 343–345. https://doi.org/10.1038/nmeth.1318 (Link opens in a new...mitochondrial genome. Nature Methods , 7(11), 901–903. https://doi.org/10.1038/nmeth.1515 (Link opens in a new...Enhancement for Gibson Isothermal Assembly. bioRxiv . https://doi.org/10.1101/2020.06.14.150979 (Link opens ...

...specificity. A good reference is this Cell paper: http://www.cell.com/abstract/S0092-8674(13)01015-5 As...plate. A good example/reference is this Cell paper: http://www.cell.com/abstract/S0092-8674(13)01016-7 In...

...can be designed using the ZiFiT Targeter software http://zifit.partners.org/ZiFiT/ChoiceMenu.aspx by choosing...

...can be designed using the ZiFiT Targeter software http://zifit.partners.org/ZiFiT/ChoiceMenu.aspx by choosing...

...RGNs and CRISPR-Cas systems can also be found at http://www.crispr-cas.org . Users are also encouraged...

...software is publicly available without registration at: http://zifit.partners.org. Links to additional pages ...

...RNAi animation Whitehead siRNA Selection Program: http://sirna.wi.mit.edu/ Back to Top I. Appendix I.1....

...more information on reagents and protocols, visit https://clover.caltech.edu. Figure adapted from Challis... Review of Neuroscience. 2022 Jul 8;45:447-469. https://doi.org/10.1146/annurev-neuro-111020-100834 Challis...in rodents. Nature Protocols;14, 379–414 (2019). https://doi.org/10.1038/s41596-018-0097-3 More resources...Using AAV-PHP.S. eNeuro,9(3), ENEURO.0373-21.2022. https://doi.org/10.1523/ENEURO.0373-21.2022 Brown, D.,...Sequencing. Frontiers in immunology, 12, 730825. https://doi.org/10.3389/fimmu.2021.730825 Campos LJ, Arokiaraj...system. Current Research in Neurobiology, 4, 100086. https://doi.org/10.1016/j.crneur.2023.100086. Challis... Annual Review of Neuroscience, 45(1), 447–469. https://doi.org/10.1146/annurev-neuro-111020-100834 Chan...

... Gene Therapy: An Overview. Cells, 12(5), 1–41. https://doi.org/10.3390/cells12050785 Murlidharan, G.,...system. Frontiers in Molecular Neuroscience, 7. https://doi.org/10.3389/fnmol.2014.00076 References and...and 9 in the Mouse Brain. PLOS ONE, 8(9), e76310. https://doi.org/10.1371/journal.pone.0076310 Damdindorj...adeno-associated viral vectors. PLoS ONE, 9(8), 1–10. https://doi.org/10.1371/journal.pone.0106472 de Backer...Hypothalamus. Human Gene Therapy, 21(6), 673–682. https://doi.org/10.1089/hum.2009.169 Dudek, A. M., Pillay...Independent of AAV Receptor. Journal of Virology, 92(7). https://doi.org/10.1128/jvi.02213-17 Favre, D., Blouin...Virus. Journal of Virology, 76(22), 11605–11611. https://doi.org/10.1128/JVI.76.22.11605-11611.2002 Griffin...

...miRFP703 https://www.addgene.org/79988/ 674 703 90,900 8.6 127 Monomer 394 4.5 37 miRFP709 https://www.addgene.org...mIFP https://www.addgene.org/54620/ 683 705 82,000 8.4 112 Monomer 54 4.5 15  15 miRFP https://www.addgene.org....0 e https://www.addgene.org/54785/ 688 709 98,000 8.1 128 Dimer 108 4.5 8  17 iRFP670 https://www.addgene.org...iRFP682 https://www.addgene.org/45459/ 663 682 90,000 11.1 162 Dimer 490 4.5 105 iRFP702 https://www.addgene.org...iRFP713 https://www.addgene.org/31857/ 690 713 98,000 6.3 100 Dimer 960 4.5 100  19 iRFP720 https://www.addgene.org...emiRFP703 https://www.addgene.org/136558/ 674 703 90,900 8.6 127 Monomer 700 4.5 90 miRFP713 https://www.addgene.org...iRFP713, % d Ref BphP-based FPs miRFP670 https://www.addgene.org/79987/ 642 670 87,400 14.0 198...

...and its host response. Nat Methods 13:868–874 . https://doi.org/10.1038/nmeth.3993  Cong L, Ran FA, Cox... Using CRISPR/Cas Systems. Science 339:819–823 .https://doi.org/10.1126/science.1231143  Cox DBT, Gootenberg...editing with CRISPR-Cas13. Science 358:1019–1027 . https://doi.org/10.1126/science.aaq0180  Fine EJ, Appleton...using compact expression cassettes. Sci Rep 5: . https://doi.org/10.1038/srep10777  Garneau JE, Dupuis ...bacteriophage and plasmid DNA. Nature 468:67–71 . https://doi.org/10.1038/nature09523  Ibraheim R, Song ...Neisseria meningitidis Cas9 in vivo. Genome Biol 19: . https://doi.org/10.1186/s13059-018-1515-0  Kim E, Koo ...derived from Campylobacter jejuni. Nat Commun 8: . https://doi.org/10.1038/ncomms14500  Levy JM, Yeh W-H,...

...conservation of a conoid. PLoS Biology 19:e3001020. https://doi.org/10.1371/journal.pbio.3001020  Chen F, ...2015) Expansion microscopy. Science 347:543–548. https://doi.org/10.1126/science.1260088 Damstra HG, Mohar...Robust Expansion Microscopy (TREx). Elife 11:e73775. https://doi.org/10.7554/elife.73775  Dani A, Huang B, ...Chemical Synapses in the Brain. Neuron 68:843–856. https://doi.org/10.1016/j.neuron.2010.11.021  Gambarotto...expansion microscopy (U-ExM). Nat Methods 16:71–74. https://doi.org/10.1038/s41592-018-0238-1  Klimas A, Gallagher... for expansion microscopy. Nat Biotechnol 1–12. https://doi.org/10.1038/s41587-022-01546-1  Mikhaylova...using anti-tubulin nanobodies. Nat Commun 6:7933. https://doi.org/10.1038/ncomms8933  M’Saad O, Bewersdorf...

... Caenorhabditis elegans. Genetics 198:837–846 . https://doi.org/10.1534/genetics.114.169730 Bottcher R...Drosophila cells. Nucleic Acids Research 42:e89–e89 . https://doi.org/10.1093/nar/gku289 Chen C, Fenk LA, de...recombination. Nucleic Acids Research 41:e193–e193 . https://doi.org/10.1093/nar/gkt805 Dickinson DJ, Pani ...Drug Selection Cassette. Genetics 200:1035–1049 . https://doi.org/10.1534/genetics.115.178335 Dokshin GA... Caenorhabditis elegans. Genetics 210:781–787 . https://doi.org/10.1534/genetics.118.301532 Ebbing A, ...alternative gene editing system for AT-rich sequences https://doi.org/10.17912/W2237D Farboud B, Severson AF...Editing Using CRISPR-Cas9. Genetics 211:431–457 . https://doi.org/10.1534/genetics.118.301775 Ghanta KS,...

...native viral promoters. Mol Ther, 7(4), 526–534. https:/doi.org/10.1016/S1525-0016(03)00023-6. PMID: 12727116...evidence. Emerg Microbes Infect, 8(1), 1679–1687. https://doi.org/10.1080/22221751.2019.1690953. PMID: 31749409...Reference Module in Biomedical Sciences. Elsevier. https://doi.org/10.1016/B978-0-12-801238-3.02523-X. Hofmann...Adenoviruses In Vivo. J Virol, 73(8), 6930–6936. https://doi.org/10.1128/JVI.73.8.6930-6936.1999. PMID:...therapy. Proc Natl Acad Sci U S A, 95(19), 11377. https://doi.org/10.1073/PNAS.95.19.11377. PMID: 9736744...Nuclear Pore Complex. PLoS Pathog, 11(6), e1004821. https://doi.org/10.1371/journal.ppat.1004821. PMID: 26042599...adenoviruses. J Clin Pathol Suppl (R Coll Pathol)., 6, 39. https://doi.org/10.1136/jcp.s3-6.1.39. PMID: 4376152. ...

...The unexhausted potential of E. coli. ELife, 4. https://doi.org/10.7554/elife.05826 Blum, M., & Ott, T...Disease. Cells Tissues Organs, 205(5–6), 303–313. https://doi.org/10.1159/000490898 Gibbs, R., Weinstock...mammalian evolution. Nature 428, 493–521 (2004). https://doi.org/10.1038/nature02426 Iannaccone, P. M.,...! Disease Models & Mechanisms, 2(5–6), 206–210. https://doi.org/10.1242/dmm.002733 Idalia, V.-M. N., &...Pathogenesis and Biotechnological Applications. InTech. https://doi.org/10.5772/67306 Koornneef, M., & Meinke,...model plant. The Plant Journal, 61(6), 909–921. https://doi.org/10.1111/j.1365-313x.2009.04086.x Krämer...ecological context with Arabidopsis thaliana. ELife, 4. https://doi.org/10.7554/elife.06100 Liu, L. S., Zhao, ...

...delivery in mice. J Clin Invest 118:1955–1964 . https://doi.org/10.1172/jci34316 Bak RO, Porteus MH (2017...Using AAV Donor Vectors. Cell Reports 20:750–756 . https://doi.org/10.1016/j.celrep.2017.06.064  Carvalho...Approaches for Retinal Targeting. Front Neurosci 11: . https://doi.org/10.3389/fnins.2017.00503  Chamberlain ...Virus Capsids. Human Gene Therapy Methods 27:1–12 . https://doi.org/10.1089/hgtb.2015.140 Frischmeyer PA (...Lacking Termination Codons. Science 295:2258–2261 . https://doi.org/10.1126/science.1067338 Ghosh A, Yue Y...Transgene-independent Manner. Molecular Therapy 16:124–130 . https://doi.org/10.1038/sj.mt.6300322 Halbert CL, Allen...parts of a larger gene. Nat Biotechnol 20:697–701 . https://doi.org/10.1038/nbt0702-697 Hirsch ML, Agbandje-McKenna...

...epigenome editing. Nucleic Acids Research 47:e23–e23. https://doi.org/10.1093/nar/gky1286 Hilton IB, D’Ippolito...and enhancers. Nature Biotechnology 33:510–517 . https://doi.org/10.1038/nbt.3199 Find plasmids from this...human genome. Nature Biotechnology 35:561–568 . https://doi.org/10.1038/nbt.3853 Find plasmids from this...-MQ1 fusion protein. Nature Communications 8: . https://doi.org/10.1038/ncomms16026 Find plasmids from...in the Mammalian Genome. Cell 167:233–247.e17 . https://doi.org/10.1016/j.cell.2016.08.056 Find plasmids...fusion proteins. Nature Biotechnology 31:1137–1142 . https://doi.org/10.1038/nbt.2726 Find plasmids from this...site-specific DNA methylation. Biology Open 5:866–874 . https://doi.org/10.1242/bio.019067 Find plasmids from...