Optimized dual promoter AAV gene therapy for lysosomal ßglucocerebrosidase with high M6P content for treatment of neuronopathic Gaucher disease

被引：0

作者：

Hedman, Andrew ^{[1
]}

Ortmeier, Steven B. ^{[2
]}

Liu, Shou ^{[1
]}

Srnak, Jennifer ^{[1
]}

Zhao, Yicheng ^{[1
]}

White, Katherine ^{[3
]}

Gotshall, Russell ^{[1
]}

Liu, Lin ^{[1
]}

Do, Hung ^{[1
]}

机构：

[1] M6P Therapeut, St Louis, MO USA

[2] Sanford Res, Sioux Falls, SD USA

[3] Sanford Hlth, Sioux Falls, SD USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2023.107876

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

137

引用

页数：1

共 5 条

[1] Novel dual promoter AAV gene therapy platform ensures production of therapeutic soluble lysosomal enzymes with high M6P content to enable broad cellular uptake and cross correction in vivo
Hedman, Andrew
Srnak, Jennifer
Zhao, Yicheng
Liu, Shou
Gotshall, Russell
Liu, Lin
Do, Hung
MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 58 - 58
[2] Novel AAV gene therapy produces beta-glucocerebrosidase with high levels of M6P to enable cellular uptake and cross-correction in the CNS as a potential treatment for type 2/3 Gaucher disease
Liu, Lin
Zhao, Yicheng
Hedman, Andrew
Srnak, Jennifer
Liu, Shou
Gotshall, Russell
Do, Hung
MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 83 - 83
[3] A novel AAV9 gene therapy for producing GCase enzyme with high mannose 6-phosphate content to treat Gaucher disease
Liu, L.
Hedman, A. C.
Srnak, J. A.
Zhao, Y.
Liu, S.
Gotschall, R. R.
Do, H.
HUMAN GENE THERAPY, 2022, 33 (23-24) : A169 - A169
[4] Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes
Massaro, Giulia
Hughes, Michael P.
Whaler, Sammie M.
Wallom, Kerri-Lee
Priestman, David A.
Platt, Frances M.
Waddington, Simon N.
Rahim, Ahad A.
HUMAN MOLECULAR GENETICS, 2020, 29 (12) : 1933 - 1949
[5] Generation of β-Glucocerebrosidase variants with increased half-life in human plasma for liver directed AAV gene therapy aimed at the treatment of Gaucher disease type 1
Comper, Fabrizio
Yu, I-Mei
Kalcheva, Petya
Miranda, Carlos J.
Chisari, Elisa
Cocita, Clement
Correia, Samantha
Pandya, Jalpa
Northcott, Natalie
Stotter, Emmaline
Shehu, Erald
Sheridan, Rose
Nathwani, Amit C.
Corbau, Romuald
MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S27 - S28

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