Liver-directed AAV gene therapy corrects disease symptoms in a murine model of lysosomal acid lipase deficiency

被引：0
|
作者：
Lam, Patricia [1 ]
Zygmunt, Deborah [1 ]
Ashbrook, Anna [1 ]
Yan, Cong [2 ]
Du, Hong [2 ]
Martin, Paul T. [1 ]
机构：
[1] Nationwide Childrens Hosp, Abigail Wexner Res Inst, Columbus, OH USA
[2] Indiana Univ Sch Med, Indianapolis, IN USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2023.107930
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
191
引用
收藏
页数：1
相关论文
共 50 条
  • [31] Liver directed in utero AAV gene therapy for maple syrup urine disease in a neonatal lethal murine model
    Mullany, Kate
    Alexander, Ian
    Siew, Susan
    JOURNAL OF HEPATOLOGY, 2022, 77 : S754 - S755
  • [32] Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapy
    Rodriguez-Marquez, Esther
    Meumann, Nadja
    Buening, Hildegard
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2021, 21 (06) : 749 - 766
  • [33] Optimization of AAV liver-directed gene therapy for the treatment of Crigler-Najjar syndrome
    Miranda, Paula
    Bakker, Conny
    Kunne, Cindy
    Bosma, Piter
    HUMAN GENE THERAPY, 2008, 19 (10) : 1079 - 1079
  • [34] Liver-directed gene targeting as a potential therapy for Fabry disease
    Saxena, H.
    Biasizzo, J.
    Domenis, R.
    Zentilin, L.
    Dardis, A.
    Muro, A. F.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A170 - A170
  • [35] Liver-Directed Gene Targeting as a Potential Therapy for Fabry Disease
    Saxena, Himanshi
    Biasizzo, Jessica
    Domenis, Rossana
    Zentilin, Lorena
    Dardis, Andrea
    Muro, Andres F.
    MOLECULAR THERAPY, 2024, 32 (04) : 733 - 733
  • [36] Liver-directed gene targeting as a potential therapy for Fabry disease
    Saxena, H.
    Biasizzo, J.
    Domenis, R.
    Zentilin, L.
    Dardis, A.
    Muro, A. F.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A241 - A241
  • [37] Preclinical Development of SPK-3006, an Investigational Liver-Directed AAV Gene Therapy for the Treatment of Pompe Disease
    Riling, Christopher R.
    Nordin, Jayme M. L.
    Verdera, Helena Costa
    Sellier, Pauline
    Preston, George Michael
    Cohen, Daniel M.
    Collaud, Fanny
    Crosariol, Marco
    Quinn, William J.
    Hanby, Hayley
    Cagin, Umut
    Puzzo, Francesco
    Haurigot, Virginia
    Ronzitti, Giuseppe
    Colella, Pasqualina
    Anguela, Xavier
    Mingozzi, Federico
    Armour, Sean M.
    MOLECULAR THERAPY, 2020, 28 (04) : 302 - 303
  • [38] First-in-human study of a liver-directed AAV gene therapy (FLT190) in Fabry disease
    Hughes, Derralynn A.
    Patel, Niten
    Kinch, Russell
    Dronfield, Leanne
    Short, Gerard
    Sheridan, Rose
    Kia, Azadeh
    Jeyakumar, Jey
    Corbau, Romuald
    Nathwani, Amit
    MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S77 - S78
  • [39] Preclinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
    Armour, Sean M.
    Nordin, Jayme M. L.
    Verdera, Helena Costa
    Sellier, Pauline
    Preston, George M.
    Riling, Christopher
    Cohen, Daniel M.
    Collaud, Fanny
    Crosariol, Marco
    Quinn, William J.
    Hanby, Hayley
    Cagin, Umut
    Puzzo, Francesco
    Haurigot, Virginia
    Ronzitti, Giuseppe
    Colella, Pasqualina
    Anguela, Xavier M.
    Mingozzi, Federico
    MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S22 - S23
  • [40] Increased Frequency of Permanent Integration Following Liver-Directed Gene Therapy with rDNA-AAV2/8 In Murine Phenylketonuria
    Winn, Shelley R.
    Cobb, Katherine E.
    Morcinek, Nick
    Roy, Laura M.
    Wang, Zhonya
    Koeberl, Dwight D.
    Grompe, Markus
    Harding, Cary O.
    MOLECULAR THERAPY, 2012, 20 : S55 - S55
12345