Skip to main content

We narrowed to 281 results for: HTT

Showing: 41 - 50 of 281 results
  1. Viral Vectors 101: Pseudotyping

    Type
    Blog Post
    ...Protein for Neutralization Assays. Viruses 12:513 . https://doi.org/10.3390/v12050513 Cronin J, Zhang X-Y,...Lentiviral Vectors through Pseudotyping. CGT 5:387–398 . https://doi.org/10.2174/1566523054546224 DePolo NJ, Reed...Inactivated by Human Serum. Molecular Therapy 2:218–222 . https://doi.org/10.1006/mthe.2000.0116 Gutierrez-Guerrero...for Research and Gene Therapy. Viruses 12:1016 . https://doi.org/10.3390/v12091016 Joglekar AV, Sandoval...Guises. Human Gene Therapy Methods 28:291–301 . https://doi.org/10.1089/hgtb.2017.084 Li Q, Liu Q, Huang...for enveloped viruses. Rev Med Virol 28:e1963 . https://doi.org/10.1002/rmv.1963 Mazarakis ND (2001) Rabies...delivery. Human Molecular Genetics 10:2109–2121 . https://doi.org/10.1093/hmg/10.19.2109 Sandrin V, Boson...
  2. Rett Syndrome: A History of Research and Therapeutic Outlooks

    Type
    Blog Post
    ...methyl-CpG-binding protein 2. Nat Genet 23:185–188 . https://doi.org/10.1038/13810 Amir RE, Zoghbi HY (2000...phenotype-genotype correlations. Am J Med Genet 97:147–152 . https://doi.org/10.1002/1096-8628(200022)97:2<147::aid-ajmg6...Syndrome and Related Disorders. Cell 152:984–996 . https://doi.org/10.1016/j.cell.2013.01.038 Guy J, Gan ...Model of Rett Syndrome. Science 315:1143–1147 . https://doi.org/10.1126/science.1138389 Le TTH, Tran NT...Human-Induced Pluripotent Stem Cells. Front Genet 10: . https://doi.org/10.3389/fgene.2019.00625 McGraw CM, Samaco... Function Requires MeCP2. Science 333:186–186 . https://doi.org/10.1126/science.1206593 Powers S, Miranda...MeCP2 null (S51.002). Neurology 92 (15 Supplement) . http://n.neurology.org/content/92/15_Supplement/S51.002...
  3. The PAM Requirement and Expanding CRISPR Beyond SpCas9

    Type
    Blog Post
    ...targeting with CRISPR–Cas13. Nature 550:280–284 . https://doi.org/10.1038/nature24049 Find plasmids from...high-fidelity genome-editing platform. Genome Biol 19: . https://doi.org/10.1186/s13059-018-1591-1 Cox DBT, Gootenberg...editing with CRISPR-Cas13. Science 358:1019–1027 . https://doi.org/10.1126/science.aaq0180 Find plasmids...endonucleases in human cells. Nat Biotechnol 34:863–868 . https://doi.org/10.1038/nbt.3609 Find plasmids from this...derived from Campylobacter jejuni. Nat Commun 8: . https://doi.org/10.1038/ncomms14500 Find plasmids from...altered PAM specificities. Nature 523:481–485 . https://doi.org/10.1038/nature14592 Find plasmids from...nucleases in human cells. Nat Biotechnol 34:869–874 . https://doi.org/10.1038/nbt.3620 Find plasmids from this...
  4. Generating Mouse Models Using CRISPR/Cas9

    Type
    Blog Post
    ...Using CRISPR/Cas Systems. Science 339:819–823 . https://doi.org/10.1126/science.1231143 Doench JG, Hartenian...gene inactivation. Nat Biotechnol 32:1262–1267 . https://doi.org/10.1038/nbt.3026 Gurumurthy CB, Takahashi...Embryos. Current Protocols in Human Genetics 88: . https://doi.org/10.1002/0471142905.hg1508s88 Leidy-Davis...CRISPR/Zygote Injection Approaches. Sci Rep 8: . https://doi.org/10.1038/s41598-018-33408-9 Mizuno N, Mizutani...Adeno-Associated Viral Vector. iScience 9:286–297 . https://doi.org/10.1016/j.isci.2018.10.030 Qin W, Kutny...Editing. Current Protocols in Mouse Biology 6:39–66 . https://doi.org/10.1002/9780470942390.mo150178 Qin W, ...Electroporation of Nuclease. Genetics 200:423–430 . https://doi.org/10.1534/genetics.115.176594 Yang H, Wang...
  5. Viral Vectors 101: Producing Your rAAV

    Type
    Blog Post
    .... Human Gene Therapy Methods, 23(1), Article 1. https://doi.org/10.1089/hgtb.2011.034 Benyamini, B., Esbin...Adeno-Associated Viral Vectors [Preprint]. Molecular Biology. https://doi.org/10.1101/2023.03.20.533580 Goodwin, M. ... Molecular Neurodegeneration, 15(1), Article 1. https://doi.org/10.1186/s13024-020-00361-z Grimm, D., ...recombinant AAV-2. Gene Therapy, 6(7), Article 7. https://doi.org/10.1038/sj.gt.3300946 Howard, D. B., &.... Human Gene Therapy Methods, 28(1), Article 1. https://doi.org/10.1089/hgtb.2016.180 Korte, J., Mienert.... Human Gene Therapy, 32(13–14), Article 13–14. https://doi.org/10.1089/hum.2020.120 Lock, M., Alvira,.... Human Gene Therapy Methods, 25(2), Article 2. https://doi.org/10.1089/hgtb.2013.131 Lock, M., McGorray...
  6. PRIDICT: Predicting Efficiencies of Prime Editing Guide RNAs

    Type
    Blog Post
    ... Integrated in Parallel. Cell, 154(4), 914–927. https://doi.org/10.1016/J.CELL.2013.07.018 Anzalone, A...breaks or donor DNA. Nature, 576(7785), 149–157. https://doi.org/10.1038/s41586-019-1711-4 Chen, P. J.,...manipulation. Nature Reviews Genetics, 24(3), Article 3. https://doi.org/10.1038/s41576-022-00541-1 Kim, H. K.,...performance. Science Advances, 5(11), eaax9249. https://doi.org/10.1126/sciadv.aax9249 Koeppel, J., Weller... determinants. Nature Biotechnology 2023, 1–11. https://doi.org/10.1038/s41587-023-01678-y Li, X., Chen...epigenetic manipulation of prime editing. Cell. https://doi.org/10.1016/j.cell.2024.03.020 Mathis, N.,... learning. Nature Biotechnology, 41, 1151–1159. https://doi.org/10.1038/s41587-022-01613-7 Mathis, N.,...
  7. "Hall of Fame" AAV Enhancers from the Allen Institute for Brain Science

    Type
    Blog Post
    ...sponsored":false,"url":{"content_id":null,"href":"https://www.addgene.org/depositor-collections/allen-institute-aav-enhancers...allen-institute-aav-enhancers/","href_with_scheme":"https://www.addgene.org/depositor-collections/allen-institute-aav-enhancers...for genetic access to cortical cell types. Cell. https://doi.org/10.1016/j.cell.2025.05.002 Hunker, A. ...perturbing striatal cell types and circuits. Neuron https://doi.org/10.1016/j.neuron.2025.04.035 Graybuck,...cell-subclass-specific labeling. Neuron, 109(9), 1449-1464.e13. https://doi.org/10.1016/j.neuron.2021.03.011 Mich, J. ...primate neocortex. Cell Reports, 34(13), 108754. https://doi.org/10.1016/j.celrep.2021.108754 Panigrahi...the known and the unknown. Genome Biology, 22(1). https://doi.org/10.1186/s13059-021-02322-1  Pennacchio...
  8. The Advances Behind The World's First Personalized CRISPR Treatment

    Type
    Blog Post
    .... Science (New York, N.Y.), 339(6121), 819–823. https://doi.org/10.1126/science.1231143  Gaudelli, N. ...without DNA cleavage. Nature, 551(7681), 464–471. https://doi.org/10.1038/nature24644  Gaudelli, N. M., ...application. Nature Biotechnology, 38(7), 892–900. https://doi.org/10.1038/s41587-020-0491-6  Komor, A. C...double-stranded DNA cleavage. Nature, 533(7603), 420–424. https://doi.org/10.1038/nature17946  Lander, E. S. (2016...2016). The Heroes of CRISPR. Cell, 164(1), 18–28. https://doi.org/10.1016/j.cell.2015.12.041  Musunuru, ...England Journal of Medicine, 392(22), 2235–2243. https://doi.org/10.1056/NEJMoa2504747  Rees, H. A., Wilson...base editors. Science Advances, 5(5), eaax5717. https://doi.org/10.1126/sciadv.aax5717  Richter, M. F....
  9. Hot Plasmids: Fall 2025

    Type
    Blog Post
    ...multicellular interactions. Neuron, 113(16), 2599–2620.e7. https://doi.org/10.1016/j.neuron.2025.05.019    Unveiling...mitochondria transfer. Nature, 623(7986), 283–291. https://doi.org/10.1038/s41586-023-06537-z Hoover, G.... during cancer metastasis. Nature, 644 252–262. https://doi.org/10.1038/s41586-025-09176-8 Spees, J. ...National Academy of Sciences USA, 103(5), 1283–1288. https://doi.org/10.1073/pnas.0510511103      Red calcium...multiplexing capabilities. bioRxiv 2024.12.16.628673. https://doi.org/10.1101/2024.12.16.628673 Zhou, S., Zhu...single-cell resolution. PLoS Biol. 23(4): e3003048. https://doi.org/10.1371/journal.pbio.3003048   Micropeptide...rare genetic diseases. Nature, 614(7948), 564–571. https://doi.org/10.1038/s41586-022-05682-1 Zhang, Y., ...
  10. Simple CRISPR-based Epigenetic Editing: dCas9-directed DNA Demethylation

    Type
    Blog Post
    ...methylated somatic enhancers. Nat Genet 52, 819–827. https://doi.org/10.1038/s41588-020-0639-9.  Feinberg A...from their normal counterparts. Nature 301, 89–92. https://doi.org/10.1038/301089a0. Galonska C, Charlton...methyltransferase footprints. Nat Commun 9, 597. https://doi.org/10.1038/s41467-017-02708-5 Hrit J, Goodrich...activity and function in development. eLife 7:e34870. https://doi.org/10.7554/eLife.34870.  Kaas GA, Zhong C... and Memory Formation. Neuron 79, 6 P1086-1093. https://doi.org/10.1016/j.neuron.2013.08.032.  Morita ...catalytic domain fusions. Nat Biotechnol 34, 1060–1065. https://doi.org/10.1038/nbt.3658. Sapozhnikov DM, Szyf...methyltransferase with CRISPR/dCas9. Nat Commun 12, 5711. https://doi.org/10.1038/s41467-021-25991-9. Spruijt CG...
Showing: 41 - 50 of 281 results