Widespread vector distribution following intra-CSF delivery of AAV9 vectors in a large animal model

被引：0
|
作者：
Haurigot, Virginia [1 ]
Marco, Sara [1 ]
Ribera, Albert [1 ]
Garcia, Miquel [1 ]
Ruzo, Albert [1 ]
Villacampa, Pilar [1 ]
Ayuso, Eduard [1 ]
Anor, Sonia [2 ]
Andaluz, Anna [2 ]
Pineda, Mercedes [3 ]
Garcia Fructoso, Gemma [3 ]
Molas, Maria [1 ]
Maggioni, Luca [1 ]
Motas, Sandra [1 ]
Ruberte, Jesus [1 ]
Mingozzi, Federico [4 ]
Pumarola, Marti [2 ]
Bosch, Fatima [1 ]
机构：
[1] Univ Autonoma Barcelona, Cbateg, E-08193 Barcelona, Spain
[2] Univ Autonoma Barcelona, Sch Vet Med, E-08193 Barcelona, Spain
[3] Hosp San Juan Dios, Barcelona, Spain
[4] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
来源：
HUMAN GENE THERAPY | 2012年 / 23卷 / 10期
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：
引用
收藏
页码：A47 / A47
页数：1
相关论文
共 20 条
  • [1] Intrathecal delivery of AAV9 vectors to model and rescue a large animal model of SMA
    Duque, S.
    Porensky, P.
    William, D. A.
    Odermatt, P.
    Nlend, R. Nlend
    Bevan, A. K.
    Foust, K.
    Braun, L.
    Schmelzer, L.
    Schumperli, D.
    Kaspar, B. K.
    Burghes, A. H. M.
    [J]. NEUROMUSCULAR DISORDERS, 2013, 23 (9-10) : 797 - 797
  • [2] Safe and Efficient Intra-CSF Delivery of AAV Vector in Nonhuman Primates
    Song, Chunjuan
    Huang, Shai
    Xu, Jiayi
    Lu, Qian
    Xin, Xin
    Zhou, Ying
    Hu, Wei
    Wang, Hui
    Wang, Lijun
    Wu, Zhenhua
    Ye, Guo-Jie
    [J]. MOLECULAR THERAPY, 2023, 31 (04) : 343 - 344
  • [3] Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
    Bey, Karim
    Deniaud, Johan
    Dubreil, Laurence
    Joussemet, Beatrice
    Cristini, Joseph
    Ciron, Carine
    Hordeaux, Juliette
    Le Boulc'h, Morwenn
    Marche, Kevin
    Maquigneau, Maud
    Guilbaud, Michael
    Moreau, Rosalie
    Larcher, Thibaut
    Deschamps, Jack-Yves
    Fusellier, Marion
    Blouin, Veronique
    Sevin, Caroline
    Cartier, Nathalie
    Adjali, Oumeya
    Aubourg, Patrick
    Moullier, Philippe
    Colle, Marie-Anne
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 17 : 771 - 784
  • [4] Superior outcomes in neuroretina following IV versus intra-CSF AAV9 gene replacement in mice with MPS IIIA
    Hemsley, Kim M.
    Winner, Leanne
    Shoubridge, Andrew
    Parkinson-Lawrence, Emma
    Lau, Adeline
    Lance, Tabitha-Rose
    King, Barbara
    Scott, William
    Snel, Marten
    Trim, Paul
    Beard, Helen
    [J]. MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [5] Evaluation of neuroretina following i.v. or intra-CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia
    Beard, Helen
    Winner, Leanne
    Shoubridge, Andrew
    Parkinson-Lawrence, Emma
    Lau, Adeline A.
    Mubarokah, Siti N.
    Lance, Tabitha-Rose
    King, Barbara
    Scott, William
    Snel, Marten F.
    Trim, Paul J.
    Hemsley, Kim M.
    [J]. CNS NEUROSCIENCE & THERAPEUTICS, 2024, 30 (08)
  • [6] Long-term efficacy following intra-CSF administration of AAV9-sulfamidase in the treatment of MPSIIIA
    Marco, S.
    Ribera, A.
    Garcia, M.
    Villacampa, P.
    Motas, S.
    Roca, C.
    Ayuso, E.
    Maggioni, L.
    Anor, S.
    Andaluz, A.
    Ruberte, J.
    Haurigot, V.
    Bosch, F.
    [J]. HUMAN GENE THERAPY, 2013, 24 (12) : A134 - A135
  • [7] Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna
    Hinderer, Christian
    Bell, Peter
    Vite, Charles H.
    Louboutin, Jean-Pierre
    Grant, Rebecca
    Bote, Erin
    Yu, Hongwei
    Pukenas, Bryan
    Hurst, Robert
    Wilson, James M.
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2014, 1 : 14051
  • [8] Broad Antibody Cross Reactions to Different AAV Serotypes in NHPs Following an Intravenous AAV9 or AAV1 Vector Delivery
    Pineda, Ricardo
    Meadows, Aaron
    Fu, Haiyan
    [J]. MOLECULAR THERAPY, 2018, 26 (05) : 106 - 106
  • [9] coAAV Vectors from the AAV-Ligand Conjugate (ALIGATERT) Platform Outperform AAV9 to Address Neurological Disorders via CSF Delivery
    Gougerot, Alexianne
    Savy, Adrien
    Thomasson, Nitza
    Kara-Ali, Kamelia
    Kunalingam, Lavaniya
    Juricek, Ludmila
    Vidal, Patrice
    Lefevre, Gaelle M.
    [J]. MOLECULAR THERAPY, 2023, 31 (04) : 526 - 526
  • [10] Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
    Ryckman, Alex E.
    Deschenes, Natalie M.
    Quinville, Brianna M.
    Osmon, Karlaina J. L.
    Mitchell, Melissa
    Chen, Zhilin
    Gray, Steven J.
    Walia, Jagdeep S.
    [J]. MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2024, 32
12