Efficacy of AAV5-GLA gene therapy in manifest Fabry disease mice

被引：0
|
作者：
Liefhebber, Jolanda M. P. [1 ]
Brasser, Giso [1 ]
Ferraz, Maria J. [2 ]
Ottenhoff, Roelof [3 ]
Efthymiopoulou, Nikoleta [1 ]
Paerels, Lieke [1 ]
Pereira, Ines [1 ]
Sonea, Andra [1 ]
Allart, Leonie [1 ]
Schwarz, Lukas K. [1 ]
Squeri, Giorgia [1 ]
Dobrynin, Greg [1 ]
Rodriguez, Rosa Crespo [1 ]
Liu, Ying Poi [1 ]
Aerts, Johannes M. F. G. [2 ]
Montenegro-Miranda, Paula S. [1 ]
机构：
[1] UniQure Biopharma BV, Amsterdam, Netherlands
[2] Leiden Univ, Leiden, Netherlands
[3] Univ Amsterdam, Amsterdam UMC, Amsterdam, Netherlands
来源：
MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2022.107206
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
223
引用
收藏
页码：81 / 82
页数：2
相关论文
共 50 条
  • [41] MOLECULAR ANALYSIS AND QUANTITATIVE DETECTION OF GLA GENE EXPRESSION VARIANTS IN PATIENTS WITH FABRY DISEASE
    Filoni, C.
    Caciotti, A.
    Carraresi, L.
    Mignani, R.
    Parini, R.
    Filocamo, M.
    Soliani, F.
    Simi, L.
    Guerrini, R.
    Zammarchi, E.
    Donati, M. A.
    Morrone, A.
    CLINICAL THERAPEUTICS, 2008, 30 : S109 - S109
  • [42] GLA Gene Mutation in Hypertrophic Cardiomyopathy with a New Variant Description: Is it Fabry's Disease?
    Chaves-Markman, Andrea Virginia
    Markman, Manuel
    Calado, Eveline Barros
    Pires, Ricardo Flores
    Oliveira Santos-Veloso, Marcelo Antonio
    Fonseca Pereira, Catarina Maria
    de Melo da Silveira Lordsleem, Andrea Bezerra
    de Lima, Sandro Goncalves
    Markman Filho, Brivaldo
    de Oliveira, Dinaldo Cavalcanti
    ARQUIVOS BRASILEIROS DE CARDIOLOGIA, 2019, 113 (01) : 77 - 83
  • [43] Future clinical and biochemical predictions of Fabry disease in females by methylation studies of the GLA gene
    Hossain, Mohammad Arif
    Wu, Chen
    Yanagisawa, Hiroko
    Miyajima, Takashi
    Akiyama, Keiko
    Eto, Yoshikatsu
    MOLECULAR GENETICS AND METABOLISM REPORTS, 2019, 20
  • [44] Mutations in the GLA Gene and LysoGb3: Is It Really Anderson-Fabry Disease?
    Duro, Giovanni
    Zizzo, Carmela
    Cammarata, Giuseppe
    Burlina, Alessandro
    Burlina, Alberto
    Polo, Giulia
    Scalia, Simone
    Oliveri, Roberta
    Sciarrino, Serafina
    Francofonte, Daniele
    Alessandro, Riccardo
    Pisani, Antonio
    Palladino, Giuseppe
    Napoletano, Rosa
    Tenuta, Maurizio
    Masarone, Daniele
    Limongelli, Giuseppe
    Riccio, Eleonora
    Frustaci, Andrea
    Chimenti, Cristina
    Ferri, Claudio
    Pieruzzi, Federico
    Pieroni, Maurizio
    Spada, Marco
    Castana, Cinzia
    Caserta, Marina
    Monte, Ines
    Rodolico, Margherita Stefania
    Feriozzi, Sandro
    Battaglia, Yuri
    Amico, Luisa
    Losi, Maria Angela
    Autore, Camillo
    Lombardi, Marco
    Zoccali, Carmine
    Testa, Alessandra
    Postorino, Maurizio
    Mignani, Renzo
    Zachara, Elisabetta
    Giordano, Antonello
    Colomba, Paolo
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2018, 19 (12)
  • [45] Functional analysis of the GLA gene promoter region and the potential impact on Fabry's disease
    Schelleckes, M.
    Lenders, M.
    Guske, K.
    Schmitz, B.
    Brand, S-M
    Brand, E.
    NAUNYN-SCHMIEDEBERGS ARCHIVES OF PHARMACOLOGY, 2012, 385 : 81 - 81
  • [46] The D313Y mutation of the GLA gene could be pathogenic for Fabry disease
    Koulousios, Konstantinos
    Pateinakis, Panagiotis
    Manou, Eleni
    Tegou, Zoi
    Triantafyllou, Angeliki
    Patsialas, Stavros
    Loules, Gedeon
    Zamanakou, Maria
    Germenis, Anastasios E.
    MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S80 - S81
  • [47] An Atypical Cardiac Manifestation of Fabry Disease from a Novel Pathological Variant on the GLA Gene
    Sarsam, Luay
    Arouni, Amy
    Haddad, Toufik Mahfood
    Onaiwu, Cherry O.
    Erickson, Christopher
    CUREUS JOURNAL OF MEDICAL SCIENCE, 2020, 12 (03)
  • [48] FABRY DISEASE: GLA GENE VARIANT ALLELES LEADING TO NORMAL ALPHA GALACTOSIDASE ACTIVITY
    Ferri, L.
    Guido, C.
    la Marca, G.
    Malvagia, S.
    Cavicchi, C.
    Fiumara, A.
    Parini, R.
    Antuzzi, D.
    Zampetti, A.
    Guerrini, R.
    Giglio, S.
    Genuardi, M.
    Donati, M. A.
    Morrone, A.
    JOURNAL OF INHERITED METABOLIC DISEASE, 2010, 33 : S129 - S129
  • [49] Description of a New GLA Gene Variant in a Patient with Hypertrophic Cardiomyopathy. Is it Fabry Disease?
    Bittencourt, Marcelo Imbroinise
    ARQUIVOS BRASILEIROS DE CARDIOLOGIA, 2019, 113 (01) : 85 - 86
  • [50] α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice
    Rodriguez-Castejon, Julen
    Alarcia-Lacalle, Ana
    Gomez-Aguado, Itziar
    Vicente-Pascual, Monica
    Solinis Aspiazu, Maria Angeles
    del Pozo-Rodriguez, Ana
    Rodriguez-Gascon, Alicia
    PHARMACEUTICS, 2021, 13 (06)
12345