Pathogenesis of Growth Failure in Murine and Canine Glycogen Storage Disease-Ia and Partial Reversal of Growth Failure Using Adeno-Associated Viral Vector Therapy

被引：0

作者：

Brooks, Elizabeth J. ^{[1
]}

Little, Dianne ^{[1
]}

Arumugam, Ramamani ^{[1
]}

Sun, Baodong ^{[1
]}

Curtis, Sarah ^{[1
,2
]}

DeMaster, Amanda ^{[1
,3
]}

Maranzano, Michael ^{[1
]}

Jackson, Mark W. ^{[4
]}

Kishnani, Priya ^{[1
]}

Freemark, Michael S. ^{[1
]}

Koeberl, Dwight D. ^{[1
]}

机构：

[1] Duke Univ, Med Ctr, Durham, NC USA

[2] Rehoboth Beach Anim Hosp, Rehoboth Beach, DE USA

[3] East Lincoln Anim Hosp, Denver, NC USA

[4] Univ Glasgow, Glasgow, Lanark, Scotland

来源：

MOLECULAR THERAPY | 2013年 / 21卷

关键词：

D O I：

暂无

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：

276

引用

页码：S105 / S106

页数：2

共 32 条

[1] Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine Glycogen Storage Disease type Ia
Brooks, Elizabeth Drake
Little, Dianne
Arumugam, Ramamani
Sun, Baodong
Curtis, Sarah
DeMaster, Amanda
Maranzano, Michael
Jackson, Mark W.
Kishnani, Priya
Freemark, Michael S.
Koeberl, Dwight D.
MOLECULAR GENETICS AND METABOLISM, 2013, 109 (02) : 161 - 170
[2] Gene therapy with adeno-associated virus (AAV) vectors in canine glycogen storage disease, type Ia.
Beaty, RM
Jackson, M
Kishnani, PS
Faulkner, E
VanCamp, S
Brown, T
Boney, A
Chen, YT
Koeberl, DD
AMERICAN JOURNAL OF HUMAN GENETICS, 2000, 67 (04) : 432 - 432
[3] Adeno-Associated Virus Gene Therapy Prevents Hepatocellular Adenoma in Murine Model of Glycogen Storage Disease Type Ia
Wang, Lili
HEPATOLOGY, 2012, 56 (05) : 1593 - 1595
[4] Global Strategy to Improve the Downstream Manufacturing Process of the Adeno-Associated Viral Vector for Glycogen Storage Disease Type Ia Treatment
Di, Wenjun
Zhou, Hanson
Chen, Dennis P.
Ren, Jack
Warren, James C.
Huang, Chao
MOLECULAR THERAPY, 2023, 31 (04) : 217 - 217
[5] Adeno-Associated Virus-Mediated Correction of a Canine Model of Glycogen Storage Disease Type Ia
Weinstein, David A.
Correia, Catherine E.
Conlon, Thomas
Specht, Andrew
Verstegen, John
Onclin-Verstegen, Karine
Campbell-Thompson, Martha
Dhaliwal, Gurmeet
Mirian, Layla
Cossette, Holly
Falk, Darin J.
Germain, Sean
Clement, Nathalie
Porvasnik, Stacy
Fiske, Laurie
Struck, Maggie
Ramirez, Harvey E.
Jordan, Juan
Andrutis, Karl
Chou, Janice Y.
Byrne, Barry J.
Mah, Cathryn S.
HUMAN GENE THERAPY, 2010, 21 (07) : 903 - 910
[6] AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia
Koeberl, Dwight D.
Pinto, Carlos
Sun, Baodong
Li, Songtao
Kozink, Daniel M.
Benjamin, Daniel K., Jr.
Demaster, Amanda K.
Kruse, Meghan A.
Vaughn, Valerie
Hillman, Steven
Bird, Andrew
Jackson, Mark
Brown, Talmage
Kishnani, Priya S.
Chen, Yuan-Tsong
MOLECULAR THERAPY, 2008, 16 (04) : 665 - 672
[7] Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
Koeberl, D. D.
Sun, B. D.
Damodaran, T. V.
Brown, T.
Millington, D. S.
Benjamin, D. J., Jr.
Bird, A.
Schneider, A.
Hillman, S.
Jackson, M.
Beaty, R. M.
Chen, Y. T.
GENE THERAPY, 2006, 13 (17) : 1281 - 1289
[8] Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
D D Koeberl
B D Sun
T V Damodaran
T Brown
D S Millington
D K Benjamin
A Bird
A Schneider
S Hillman
M Jackson
R M Beaty
Y T Chen
Gene Therapy, 2006, 13 : 1281 - 1289
[9] Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
D D Koeberl
B D Sun
T V Damodaran
T Brown
D S Millington
D K Benjamin
A Bird
A Schneider
S Hillman
M Jackson
R M Beaty
Y T Chen
Gene Therapy, 2007, 14 : 281 - 281
[10] Erratum: Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
D D Koeberl
B D Sun
T V Damodaran
T Brown
D S Millington
D K Benjamin
A Bird
A Schneider
S Hillman
M Jackson
R M Beaty
Y T Chen
Gene Therapy, 2006, 13 : 1430 - 1430

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