Tongue and Hypoglossal Motoneuron Gene Therapy in a Rat Model of Pompe Disease

被引：1
作者：
Singer, Michele
Rana, Sabhya
Pope, Megan
Patterson, LiAna
Corti, Manuela
Byrne, Barry
Fuller, David
机构：
来源：
FASEB JOURNAL | 2021年 / 35卷
关键词：
D O I：
10.1096/fasebj.2021.35.S1.02822
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
引用
收藏
页数：2
相关论文
共 50 条
[11]   Lentiviral gene therapy for Pompe disease [J].
Stok, M. ;
de Boer, H. ;
de Visser, T. P. ;
Reuser, A. J. ;
Jacobs, E. ;
Jahr, H. ;
Duncker, D. ;
van Til, N. P. ;
Wagemaker, G. .
HUMAN GENE THERAPY, 2011, 22 (10) :A40-A41
[12]   Accessible Gene Therapy for Pompe Disease [J].
Catalano, F. ;
Vlaar, E. C. ;
Zundo, G. ;
Liang, Q. ;
Huizer, T. F. ;
van der Ploeg, A. T. ;
Gordaliza, I. ;
Asseldonk, D. ;
Beek, N. ;
van den Hout, H. J. M. P. ;
Schaaf, G. ;
Pijnappel, P. W. W. M. .
HUMAN GENE THERAPY, 2025, 36 (3-4) :E551-E552
[13]   Gene Therapy Developments for Pompe Disease [J].
Unnisa, Zeenath ;
Yoon, John K. ;
Schindler, Jeffrey W. ;
Mason, Chris ;
van Til, Niek P. .
BIOMEDICINES, 2022, 10 (02)
[14]   Tongue weakness in Pompe disease [J].
Dubrovsky, A. ;
Corderi, J. C. .
NEUROMUSCULAR DISORDERS, 2008, 18 (9-10) :803-803
[15]   MODULATION OF NEONATAL RAT HYPOGLOSSAL MOTONEURON EXCITABILITY BY SEROTONIN [J].
BERGER, AJ ;
BAYLISS, DA ;
VIANA, F .
NEUROSCIENCE LETTERS, 1992, 143 (1-2) :164-168
[16]   Developmental changes in hypoglossal motoneuron morphology in neonatal rat [J].
Williams, Paul Allen ;
Bellinger, Denise ;
Wilson, Christopher G. .
FASEB JOURNAL, 2018, 32 (01)
[17]   Treatment of Infantile-Onset Pompe Disease in a Rat Model with Muscle-Directed AAV Gene Therapy [J].
Garcia, Miquel ;
Munoz, Sergio ;
Bertolin, Joan ;
Jimenez, Veronica ;
Jaen, Maria Luisa ;
Pujol, Anna ;
Vila, Laia ;
Sacristan, Victor ;
Barbon, Elena ;
Ronzitti, Giuseppe ;
El Andari, Jihad ;
Tulalamba, Warut ;
Pham, Quang Hong ;
Ruberte, Jesus ;
Vandendriessche, Thierry C. ;
Chuah, Marinee K. L. ;
Grimm, Dirk ;
Mingozzi, Federico ;
Bosch, Fatima .
MOLECULAR THERAPY, 2023, 31 (04) :785-785
[18]   Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy [J].
Munoz, Sergio ;
Bertolin, Joan ;
Jimenez, Veronica ;
Jaen, Maria Luisa ;
Garcia, Miquel ;
Pujol, Anna ;
Vila, Laia ;
Sacristan, Victor ;
Barbon, Elena ;
Ronzitti, Giuseppe ;
El Andari, Jihad ;
Tulalamba, Warut ;
Pham, Quang Hong ;
Ruberte, Jesus ;
VandenDriessche, Thierry ;
Chuah, Marinee K. ;
Grimm, Dirk ;
Mingozzi, Federico ;
Bosch, Fatima .
MOLECULAR METABOLISM, 2024, 81
[19]   Progress and challenges of gene therapy for Pompe disease [J].
Ronzitti, Giuseppe ;
Collaud, Fanny ;
Laforet, Pascal ;
Mingozzi, Federico .
ANNALS OF TRANSLATIONAL MEDICINE, 2019, 7 (13)
[20]   Gene-cell therapy for Pompe disease [J].
Gonzalez-Correa, J. E. ;
Aguilar-Gonzalez, A. ;
Munoz, P. ;
Martin, F. .
HUMAN GENE THERAPY, 2019, 30 (11) :A154-A154
12345