Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System

被引：5
作者：
McElroy, Abigail [1 ,2 ]
Sena-Esteves, Miguel [1 ,2 ,3 ]
Arjomandnejad, Motahareh [1 ,2 ]
Keeler, Allison M. M. [1 ,2 ,4 ]
Gray-Edwards, Heather L. L. [1 ,2 ,5 ,6 ,7 ]
机构：
[1] UMass Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA USA
[2] UMass Chan Med Sch, Li Weibo Inst Rare Dis Res, Worcester, MA USA
[3] UMass Chan Med Sch, Dept Neurol, Worcester, MA USA
[4] UMass Chan Med Sch, Dept Pediat, Worcester, MA USA
[5] UMass Chan Med Sch, Dept Radiol, Worcester, MA USA
[6] UMass Chan Med Sch, Horae Gene Therapy Ctr, 368 Plantat St, Worcester, MA 01605 USA
[7] UMass Chan Med Sch, Li Weibo Inst Rare Dis Res, 368 Plantat St, Worcester, MA 01605 USA
来源：
HUMAN GENE THERAPY | 2022年 / 33卷 / 17-18期
关键词：
central nervous system; gene therapy; AAV; NEUTRALIZING ANTIBODIES; TRANSGENE EXPRESSION; SKELETAL-MUSCLE; ADULT MICE; BRAIN; AAV; VECTORS; DELIVERY; TURNOVER; ROUTES;
D O I：
10.1089/hum.2022.170
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：
[No abstract available]
引用
收藏
页码：889 / 892
页数：4
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共 41 条
  • [1] Evaluating Readministration of Adeno-Associated Virus for Gene Therapy
    Arjomandnejad, Motahareh
    Keeler, Allison M.
    [J]. HUMAN GENE THERAPY, 2022, 33 (5-6) : 218 - 220
  • [2] Lysosomal storage diseases and the blood-brain barrier
    Begley, David J.
    Pontikis, Charles C.
    Scarpa, Maurizio
    [J]. CURRENT PHARMACEUTICAL DESIGN, 2008, 14 (16) : 1566 - 1580
  • [3] Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
    Bertin, Berangere
    Veron, Philippe
    Leborgne, Christian
    Deschamps, Jack-Yves
    Moullec, Sophie
    Fromes, Yves
    Collaud, Fanny
    Boutin, Sylvie
    Latournerie, Virginie
    van Wittenberghe, Laetitia
    Delache, Benoit
    Le Grand, Roger
    Dereuddre-Bosquet, Nathalie
    Benveniste, Olivier
    Moullier, Philippe
    Masurier, Carole
    Merten, Otto
    Mingozzi, Federico
    [J]. SCIENTIFIC REPORTS, 2020, 10 (01)
  • [4] Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
    Bevan, Adam K.
    Duque, Sandra
    Foust, Kevin D.
    Morales, Pablo R.
    Braun, Lyndsey
    Schmelzer, Leah
    Chan, Curtis M.
    McCrate, Mary
    Chicoine, Louis G.
    Coley, Brian D.
    Porensky, Paul N.
    Kolb, Stephen J.
    Mendell, Jerry R.
    Burghes, Arthur H. M.
    Kaspar, Brian K.
    [J]. MOLECULAR THERAPY, 2011, 19 (11) : 1971 - 1980
  • [5] 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
  • [6] Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
    Boutin, Sylvie
    Monteilhet, Virginie
    Veron, Philippe
    Leborgne, Christian
    Benveniste, Olivier
    Montus, Marie Francoise
    Masurier, Carole
    [J]. HUMAN GENE THERAPY, 2010, 21 (06) : 704 - 712
  • [7] Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back
    Buscara, Laurine
    Gross, David-Alexandre
    Daniele, Nathalie
    [J]. JOURNAL OF PERSONALIZED MEDICINE, 2020, 10 (04): : 1 - 44
  • [8] Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
    Chand, Deepa H.
    Zaidman, Craig
    Arya, Kapil
    Millner, Rachel
    Farrar, Michelle A.
    Mackie, Fiona E.
    Goedeker, Natalie L.
    Dharnidharka, Vikas R.
    Dandamudi, Raja
    Reyna, Sandra P.
    [J]. JOURNAL OF PEDIATRICS, 2021, 231 : 265 - 268
  • [9] Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Tai, Chun-Hwei
    Muramatsu, Shin-ichi
    Byrne, Barry J.
    Hwu, Wuh-Liang
    [J]. LANCET CHILD & ADOLESCENT HEALTH, 2017, 1 (04) : 265 - 273
  • [10] Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses
    Ciesielska, Agnieszka
    Hadaczek, Piotr
    Mittermeyer, Gabriele
    Zhou, Shangzhen
    Wright, J. Fraser
    Bankiewicz, Krystof S.
    Forsayeth, John
    [J]. MOLECULAR THERAPY, 2013, 21 (01) : 158 - 166
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