AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset D Goertsen, NC Flytzanis, N Goeden, MR Chuapoco, A Cummins, Y Chen, ... Nature Neuroscience 25 (1), 106-115, 2022 | 297 | 2022 |
Adeno-associated virus toolkit to target diverse brain cells RC Challis, S Ravindra Kumar, X Chen, D Goertsen, GM Coughlin, ... Annual review of neuroscience 45 (1), 447-469, 2022 | 105 | 2022 |
Functional gene delivery to and across brain vasculature of systemic AAVs with endothelial-specific tropism in rodents and broad tropism in primates X Chen, DA Wolfe, DS Bindu, M Zhang, N Taskin, D Goertsen, TF Shay, ... Nature communications 14 (1), 3345, 2023 | 44 | 2023 |
Primate-conserved carbonic anhydrase IV and murine-restricted LY6C1 enable blood-brain barrier crossing by engineered viral vectors TF Shay, EE Sullivan, X Ding, X Chen, S Ravindra Kumar, D Goertsen, ... Science advances 9 (16), eadg6618, 2023 | 25 | 2023 |
Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid D Goertsen, N Goeden, NC Flytzanis, V Gradinaru Molecular Therapy Methods & Clinical Development 26, 331-342, 2022 | 23 | 2022 |
Broad gene expression throughout the mouse and marmoset brain after intravenous delivery of engineered AAV capsids NC Flytzanis, N Goeden, D Goertsen, A Cummins, J Pickel, V Gradinaru BioRxiv, 2020.06. 16.152975, 2020 | 20 | 2020 |
Primate-conserved Carbonic Anhydrase IV and murine-restricted Ly6c1 are new targets for crossing the blood-brain barrier TF Shay, EE Sullivan, X Ding, X Chen, SR Kumar, D Goertsen, D Brown, ... bioRxiv, 2023.01. 12.523632, 2023 | 4 | 2023 |
CAP-002: Systemic AAV Gene Therapy with Next Generation Capsids for Treatment of STXBP1 Encephalopathy A Knoll, AJ Lopez, T Sandberg, B Wheeler, K Whitley, LS Grigoryeva, ... MOLECULAR THERAPY 32 (4), 268-268, 2024 | | 2024 |
Parallel Engineering and Recombination of Adeno-Associated Virus Variable Regions Enables Multisite Library Production D Goertsen, V Gradinaru MOLECULAR THERAPY 31 (4), 21-21, 2023 | | 2023 |
Expanding adeno-associated viral capsid engineering to multiple variable regions for diversified tropism DG Goertsen California Institute of Technology, 2023 | | 2023 |
Transduction Profiles of Engineered Adeno-Associated Viral Capsids in Mouse and Marmoset D Goertsen, NC Flytzanis, N Goeden, A Cummins, J Pickel, V Gradinaru MOLECULAR THERAPY 28 (4), 269-270, 2020 | | 2020 |