60 Matching Annotations
  1. Oct 2026
  2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Self-inactivating AAV-CRISPR at different ages enables sustained amelioration of Huntington’s disease deficits in BAC226Q mice
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12998519/
  3. Aug 2026
  4. link.springer.com link.springer.com
    Distribution of transgene in the rodent choroid plexus after intracerebroventricular injection of adeno-associated virus
    1
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    link.springer.com/article/10.1186/s12987-026-00831-4
  5. Jun 2026
  6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    CRISPR-Cpf1-Mediated Gene-Editing System Based on a Single Bidirectional Promoter
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC13206784/
  7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Treatment of Huntington’s disease with a pan-HTT-targeting CRISPR nuclease
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC13197975/
  8. Feb 2026
  9. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Regulatable In Vivo Gene Expression via Adaptamers
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12767312/
  10. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Combination of Cas9 and adeno-associated vectors enables efficient in vivo knockdown of precise miRNAs in the rodent and primate brain
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12718335/
  11. Jan 2026
  12. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Treatment of Huntington’s disease with a pan-HTT-targeting CRISPR nuclease
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12776121/
  13. Nov 2025
  14. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Enhanced cleavage of genomic CCR5 using CASX2Max
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12562720/
  15. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    PAM-readID is a rapid, simple, and accurate PAM determination method for CRISPR-Cas enzymes in mammalian cells
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12627570/
  16. Sep 2025
  17. www.sciencedirect.com www.sciencedirect.com
    Ultrabright chemical labeling enables rapid neural connectivity profiling in large tissue samples
    1
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    sciencedirect.com/science/article/pii/S0896627325006579
  18. Aug 2025
  19. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    ENHANCED CLEAVAGE OF GENOMIC CCR5 USING CASX2Max
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    pmc.ncbi.nlm.nih.gov/articles/PMC12265720/
  20. Jul 2025
  21. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Protocol for CRISPR-based manipulation and visualization of endogenous α-synuclein in cultured mouse hippocampal neurons
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12305205/
  22. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Focused ultrasound‐mediated APOE4 knockdown in mouse brain
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12263348/
  23. www.biorxiv.org www.biorxiv.org
    Engineering a human-based translational activator for targeted protein expression restoration
    1
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    biorxiv.org/content/10.1101/2025.07.09.663984v1
  24. Jun 2025
  25. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11386987/
  26. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    CRISPR antiviral inhibits neurotrophic JC polyomavirus in 2D and 3D culture models through dual-gRNA excision by SaCas9
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC12159223/
  27. Apr 2025
  28. physoc.onlinelibrary.wiley.com physoc.onlinelibrary.wiley.com
    Neuron–non-neuron electrical coupling networks are involved in chronic stress-induced electrophysiological changes in lateral habenular neurons
    1
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    physoc.onlinelibrary.wiley.com/doi/10.1113/JP287286
  29. Mar 2025
  30. www.sciencedirect.com www.sciencedirect.com
    Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy
    1
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    sciencedirect.com/science/article/pii/S2666379125001107
  31. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Non-invasive detection of allele-specific CRISPR-SaCas9-KKH disruption of TOR1A DYT1 allele in a xenograft mouse model
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11925580/
  32. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Molecularly distinct striatonigral neuron subtypes differentially regulate locomotion
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11923167/
  33. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Electroporation Induces Unexpected Alterations in Gene Expression: A Tip for Selection of Optimal Transfection Method
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11854308/
  34. Feb 2025
  35. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Optimal SpCas9- and SaCas9-mediated gene editing by enhancing gRNA transcript levels through scaffold poly-T tract reduction
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11823040/
  36. Jan 2025
  37. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Rational engineering of minimally immunogenic nucleases for gene therapy
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11696374/
  38. Dec 2024
  39. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    All-in-one AAV-mediated Nrl gene inactivation rescues retinal degeneration in Pde6a mice
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11665581/
  40. www.biorxiv.org www.biorxiv.org
    A Versatile Anti-CRISPR Platform for Opto- and Chemogenetic Control of CRISPR-Cas9 and Cas12 across a Wide Range of Orthologs
    1
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    biorxiv.org/content/10.1101/2024.11.25.625186v1
  41. Nov 2024
  42. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Patch and matrix striatonigral neurons differentially regulate locomotion
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC11230471/
  43. Aug 2024
  44. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Suppression of HSV-1 infection and viral reactivation by CRISPR-Cas9 gene editing in 2D and 3D culture models
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11339036/
  45. www.cell.com www.cell.com
    Upregulation of utrophin improves the phenotype of Duchenne muscular dystrophy hiPSC-derived CMs
    1
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    cell.com/molecular-therapy-family/nucleic-acids/abstract/S2162-2531(24)00134-3
  46. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10422012/
  47. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC7616320/
  48. Jul 2024
  49. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Long term rescue of Alzheimer’s deficits in vivo by one-time gene-editing of App C-terminus
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11185791/
  50. Jun 2024
  51. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Conditional knockout of Shank3 in the ventral CA1 by quantitative in vivo genome-editing impairs social memory in mice
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11169449/
  52. 52.53.155.43 52.53.155.43
    Optimization of AAV vectors for transactivator-regulated enhanced gene expression within targeted neuronal populations.
    1
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    52.53.155.43/7f9ee4f71b8a925e453a4463d9dde20f671e0e7e/PIIS2589004224011003-html.html
  53. May 2024
  54. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    In vivo genome editing via CRISPR/Cas9-mediated homology-independent targeted integration for Bietti crystalline corneoretinal dystrophy treatment
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC11074121/
  55. Mar 2024
  56. 52.53.155.43 52.53.155.43
    Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1
    2
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    52.53.155.43/b4a5d5f166b97df7aae394dba3752d5bbdf7ed06/torella-et-al-2024-efficient-and-safe-therapeutic-use-of-paired-cas9-nickases-for-primary-hyperoxaluria-type-1-html.html
  57. Nov 2023
  58. www.sciencedirect.com www.sciencedirect.com
    Unexpected extra exon skipping in the DYSF gene during restoring the reading frame by CRISPR/Cas9
    1
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    sciencedirect.com/science/article/pii/S0303264723002472
  59. Sep 2023
  60. www.mdpi.com www.mdpi.com
    Targeted Delivery of Chimeric Antigen Receptor into T Cells via CRISPR-Mediated Homology-Directed Repair with a Dual-AAV6 Transduction System
    1
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    mdpi.com/1467-3045/45/10/486
  61. Aug 2023
  62. www.sciencedirect.com www.sciencedirect.com
    Neural mechanisms underlying uninstructed orofacial movements during reward-based learning behaviors
    1
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    sciencedirect.com/science/article/pii/S096098222300920X
  63. Jul 2023
  64. onlinelibrary.wiley.com onlinelibrary.wiley.com
    Production of adeno-associated viral vector serotype 6 by triple transfection of suspension HEK293 cells at higher cell densities
    1
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    onlinelibrary.wiley.com/doi/10.1002/biot.202300051
  65. May 2023
  66. www.authorea.com www.authorea.com
    High-cell-density production of adeno-associated viral vector serotype 6 by triple transfection in suspension HEK293 cell cultures
    1
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    authorea.com/users/582383/articles/622468-high-cell-density-production-of-adeno-associated-viral-vector-serotype-6-by-triple-transfection-in-suspension-hek293-cell-cultures
  67. Apr 2023
  68. www.biorxiv.org www.biorxiv.org
    Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs
    1
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    biorxiv.org/content/10.1101/2023.04.13.536294v1
  69. Mar 2023
  70. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Correction of F8 intron 1 inversion in hemophilia A patient-specific iPSCs by CRISPR/Cas9 mediated gene editing
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC10033665/
  71. Dec 2022
  72. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC9664411/
  73. Sep 2022
  74. www.sciencedirect.com www.sciencedirect.com
    Generation of a homozygous P4HA2 knockout human embryonic stem cell line (FDCHDPe012-A) by CRISPR/Cas9 system
    1
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    sciencedirect.com/science/article/pii/S1873506122002793
  75. Jun 2022
  76. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Deficiency in endocannabinoid synthase DAGLB contributes to early onset Parkinsonism and murine nigral dopaminergic neuron dysfunction
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC9205912/
  77. Mar 2022
  78. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov
    Regulation of GTPase function by autophosphorylation
    1
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    pmc.ncbi.nlm.nih.gov/articles/PMC8986090/
  79. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Antiviral Targeting of Varicella Zoster Virus Replication and Neuronal Reactivation Using CRISPR/Cas9 Cleavage of the Duplicated Open Reading Frames 62/71
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC8880005/
  80. Dec 2021
  81. elifesciences.org elifesciences.org
    scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution
    2
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    elifesciences.org/articles/64175
  82. Oct 2021
  83. www.sciencedirect.com www.sciencedirect.com
    Bombesin-like peptide recruits disinhibitory cortical circuits and enhances fear memories
    1
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    sciencedirect.com/science/article/pii/S0092867421010606
  84. Sep 2021
  85. www.ncbi.nlm.nih.gov www.ncbi.nlm.nih.gov
    Unlocking loxP to Track Genome Editing In Vivo
    1
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    ncbi.nlm.nih.gov/pmc/articles/PMC8394901/
  86. Aug 2021
  87. www.sciencedirect.com www.sciencedirect.com
    Generation of a homozygous LRPAP1 knockout human embryonic stem cell line (FDCHDPe009-B) by CRISPR/Cas9 system
    1
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    sciencedirect.com/science/article/pii/S1873506121003639
  88. www.mdpi.com www.mdpi.com
    Adeno-Associated Vector-Delivered CRISPR/SaCas9 System Reduces Feline Leukemia Virus Production In Vitro
    1
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    mdpi.com/1999-4915/13/8/1636/htm
  89. www.jneurosci.org www.jneurosci.org
    Subretinal Human Umbilical Tissue-Derived Cell Transplantation Preserves Retinal Synaptic Connectivity and Attenuates Müller Glial Reactivity
    1
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    jneurosci.org/content/38/12/2923
  90. www.sciencedirect.com www.sciencedirect.com
    CRISPR-READI: Efficient Generation of Knockin Mice by CRISPR RNP Electroporation and AAV Donor Infection
    1
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    sciencedirect.com/science/article/pii/S2211124719307429
  91. Apr 2021
  92. www.sciencedirect.com www.sciencedirect.com
    Generation of a homozygous LRP2 knockout human embryonic stem cell line (FDCHDPe010-A-56) by CRISPR/Cas9 system
    1
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    sciencedirect.com/science/article/pii/S1873506121001884
  93. Sep 2020
  94. www.jneurosci.org www.jneurosci.org
    Dopaminergic signaling in the nucleus accumbens modulates stress-coping strategies during inescapable stress
    1
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    jneurosci.org/content/40/38/7241
  95. www.sciencedirect.com www.sciencedirect.com
    The Raphe Dopamine System Controls the Expression of Incentive Memory
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    sciencedirect.com/science/article/pii/S0896627320301082
  96. Jan 2020
  97. www.sciencedirect.com www.sciencedirect.com
    Programmable RNA-Guided RNA Effector Proteins Built from Human Parts
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    sciencedirect.com/science/article/pii/S0092867419306208