The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease

P.17.8 The co-formulation of pharmacological chaperone AT2220 with recombinant human acid alpha-glucosidase improves enzyme uptake and glycogen reduction in a mouse model of Pompe disease

Neuromuscular Disorders ◽

10.1016/j.nmd.2013.06.658 ◽

2013 ◽

Vol 23 (9-10) ◽

pp. 828-829

Author(s):

R. Khanna ◽

S. Xu ◽

L. Pellegrino ◽

Y. Lun ◽

R. Soska ◽

...

Keyword(s):

Mouse Model ◽

Pompe Disease ◽

Human Acid ◽

M.P.5.09 Pharmacological chaperone therapy for the treatment of Pompe disease: Deoxynojirimycin increases lysosomal levels and specific activity of acid alpha-glucosidase

Neuromuscular Disorders ◽

10.1016/j.nmd.2007.06.426 ◽

2007 ◽

Vol 17 (9-10) ◽

pp. 889-890

Author(s):

B. Wustman ◽

A. Powe ◽

J. Flanagan ◽

R. Khanna ◽

W. Liang ◽

...

Keyword(s):

Pompe Disease ◽

Specific Activity ◽

G.P.8.05 The pharmacological chaperone AT2220 increases mutant acid alpha-glucosidase levels and reduces tissue glycogen in a mouse model of Pompe disease

Neuromuscular Disorders ◽

10.1016/j.nmd.2009.06.155 ◽

2009 ◽

Vol 19 (8-9) ◽

pp. 592 ◽

Cited By ~ 1

Author(s):

R. Khanna ◽

R. Soksa ◽

J. Feng ◽

Y. Lun ◽

A.C. Powe ◽

...

Keyword(s):

Mouse Model ◽

Pompe Disease ◽

Transgenic mouse model for familial amyotrophic lateral sclerosis with superoxide dismutase-1 mutation

Neuropathology ◽

10.1046/j.1440-1789.2001.00361.x ◽

2001 ◽

Vol 21 (1) ◽

pp. 82-92 ◽

Cited By ~ 72

Author(s):

Noriyuki Shibata

Keyword(s):

Amyotrophic Lateral Sclerosis ◽

Superoxide Dismutase ◽

Mouse Model ◽

Transgenic Mouse ◽

Transgenic Mouse Model ◽

Familial Amyotrophic Lateral Sclerosis ◽

Superoxide Dismutase 1 ◽

Lateral Sclerosis

Review for "Humanized CD22 transgenic mouse model for in vivo analysis of anti‐CD22‐based immunotherapy"

10.1002/eji.202048636/v2/review1 ◽

2020 ◽

Author(s):

Matthew Macauley

Keyword(s):

Mouse Model ◽

Transgenic Mouse ◽

Transgenic Mouse Model

Review for "Humanized CD22 transgenic mouse model for in vivo analysis of anti‐CD22‐based immunotherapy"

10.1002/eji.202048636/v1/review1 ◽

2020 ◽

Keyword(s):

Mouse Model ◽

Transgenic Mouse ◽

Transgenic Mouse Model

Increased Expression of Human Cu-Zn Superoxide Dismutase in a Transgenic Mouse Model of Ischemia/reperfusion

Journal of the American College of Cardiology ◽

10.1016/s0735-1097(97)85421-6 ◽

1998 ◽

Vol 31 (2) ◽

pp. 395A

Author(s):

J Teerlink

Keyword(s):

Superoxide Dismutase ◽

Mouse Model ◽

Transgenic Mouse ◽

Ischemia Reperfusion ◽

Transgenic Mouse Model

Spontaneous Chronic Liver Inflammation in a Transgenic Mouse Model of Autoimmune Hepatitis: Impact and Regulation of Autoreactive CD8+ T Cells

Zeitschrift für Gastroenterologie ◽

10.1055/s-0029-1191959 ◽

2009 ◽

Vol 47 (01) ◽

Author(s):

M Zierden ◽

E Kühnen ◽

M Odenthal ◽

HP Dienes

Keyword(s):

T Cells ◽

Mouse Model ◽

Autoimmune Hepatitis ◽

Transgenic Mouse ◽

Cd8 T Cells ◽

Transgenic Mouse Model ◽

Chronic Liver ◽

Liver Inflammation

Diffusion kurtosis imaging detects a very early α-synuclein accumulation induced changes in transgenic mouse model of parkinson’s disease overexpressing α-synuclein

10.26226/morressier.5785edc7d462b80296c994db ◽

2016 ◽

Author(s):

Anas Arab

Keyword(s):

Parkinson’S Disease ◽

Parkinson's Disease ◽

Mouse Model ◽

Transgenic Mouse ◽

Transgenic Mouse Model ◽

Diffusion Kurtosis Imaging ◽

Induced Changes ◽

Diffusion Kurtosis

Faculty Opinions recommendation of Preserved fronto-striatal plasticity and enhanced procedural learning in a transgenic mouse model of Alzheimer's disease overexpressing mutant hAPPswe.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.1020559.235469 ◽

2004 ◽

Author(s):

Karl-Peter Giese

Keyword(s):

Alzheimer’S Disease ◽

Alzheimer's Disease ◽

Mouse Model ◽

Transgenic Mouse ◽

Transgenic Mouse Model ◽

Procedural Learning ◽

Model Of Alzheimer’S Disease