Targeting AAV Vectors to the CNS via De Novo Engineered Capsid-Receptor Interactions

被引:0
|
作者
Huang, Qin [1 ]
Chen, Albert [2 ]
Chan, Ken Y. [1 ]
Sorensen, Hikari [1 ]
Barry, Andrew J. [1 ]
Azari, Bahar [3 ]
Beddow, Thomas [1 ]
Zheng, Qingxia [1 ]
Zhao, Binhui [1 ]
Tobey, Isabelle G. [1 ]
Eid, Fatma Elzahraa [1 ]
Chan, Alina [1 ]
Deverman, Benjamin E. [1 ]
机构
[1] Broad Inst MIT & Harvard, Cambridge, MA USA
[2] Harvard Univ, Cambridge, MA USA
[3] Northeastern Univ, Elect & Comp Engn Dept, Boston, MA USA
关键词
D O I
暂无
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
441
引用
收藏
页码:228 / 229
页数:2
相关论文
共 50 条
  • [1] Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier
    Huang, Qin
    Chen, Albert T. J.
    Chan, Ken Y.
    Sorensen, Hikari
    Barry, Andrew
    Azari, Bahar
    Zheng, Qingxia G.
    Beddow, Thomas
    Zhao, Binhui
    Tobey, Isabelle J.
    Moncada-Reid, Cynthia
    Eid, Fatma-Elzahraa R.
    Walkey, Christopher D.
    Ljungberg, M. Cecilia A.
    Lagor, William E.
    Heaney, Jason
    Chan, Yujia
    Deverman, Benjamin
    PLOS BIOLOGY, 2023, 21 (07)
  • [2] AAV-Mediated CNS-Wide Gene Delivery via a De Novo Engineered Capsid-Human Receptor Interaction
    Chan, Ken Y.
    Huang, Qin
    Johnston, Jencilin
    Barry, Andrew J.
    Zheng, Qingxia
    Chen, Albert T.
    Beddow, Thomas
    Keyes, Casey
    Pacouret, Simon
    Brauer, Pamela P.
    Lou, Shan
    Clouse, Gabrielle
    Wu, Jason
    Navarrete, Nuria R. R.
    Eid, Fatma Elzahraa
    Chan, Yujia A.
    Deverman, Benjamin E.
    MOLECULAR THERAPY, 2023, 31 (04) : 56 - 56
  • [3] In Vivo targeting of HSC by capsid-engineered AAV vectors
    Meumann, N.
    Radtke, S.
    Garmendia, X. Perez
    Just, S.
    Gutierrez, A. Fornell
    Ertelt, M.
    Schoeder, C. T.
    Kuhlmann, A. S.
    Peterson, C. W.
    Stone, D.
    Jerome, K.
    Kiem, H. P.
    Buening, H.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A9 - A10
  • [4] Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
    Buning, Hildegard
    Srivastava, Arun
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2019, 12 : 248 - 265
  • [5] Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors
    Kay, Christine N.
    Ryals, Renee C.
    Aslanidi, George V.
    Min, Seok Hong
    Ruan, Qing
    Sun, Jingfen
    Dyka, Frank M.
    Kasuga, Daniel
    Ayala, Andrea E.
    Van Vliet, Kim
    Agbandje-McKenna, Mavis
    Hauswirth, William W.
    Boye, Sanford L.
    Boye, Shannon E.
    PLOS ONE, 2013, 8 (04):
  • [6] Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid
    Martino, R. Alexander
    Wang, Qiang
    Xu, Hao
    Hu, Gui
    Bell, Peter
    Arroyo, Edgardo. J. J.
    Sims, Joshua. J. J.
    Wilson, James. M. M.
    JOURNAL OF VIROLOGY, 2023, 97 (06) : e0017423
  • [7] Engineered AAV capsid transport mutants overcome transduction deficiencies in the aged CNS
    Sandoval, Ivette M.
    Kelley, Christy M.
    Bernal-Conde, Luis Daniel
    Steece-Collier, Kathy
    Marmion, David J.
    Davidsson, Marcus
    Crosson, Sean M.
    Boye, Sanford L.
    Boye, Shannon E.
    Manfredsson, Fredric P.
    MOLECULAR THERAPY NUCLEIC ACIDS, 2024, 35 (04):
  • [8] Cell infection by capsid engineered AAV vectors: variations from the common scheme?
    Uhrig, Silke
    Perabo, Luca
    Hallek, Michael
    Buening, Hildegard
    HUMAN GENE THERAPY, 2008, 19 (10) : 1191 - 1191
  • [9] The Capsid Race: Multiplexed Analysis and Comparison of Rationally Engineered AAV CNS Capsids In Vivo
    Pimentel, Elena
    Kieper, Sebastian
    Moreno, Fiona
    Van der Zon, Tom
    Bosma, Bas
    Bashir, Noun
    Waaijer, Stijn
    Espinosa, Pedro
    Hoogland, Tycho
    Valls, Mercedes
    Pesic, Milja
    Kusuma, Angga
    MOLECULAR THERAPY, 2023, 31 (04) : 231 - 232
  • [10] Identification and validation of novel engineered AAV capsid variants targeting human glia
    Giacomoni, Jessica
    Akerblom, Malin
    Habekost, Mette
    Fiorenzano, Alessandro
    Kajtez, Janko
    Davidsson, Marcus
    Parmar, Malin
    Bjorklund, Tomas
    FRONTIERS IN NEUROSCIENCE, 2024, 18