Fatigue and motor gains in children with Pompe disease undergoing gene replacement therapy for chronic ventilatory failure

2013 ◽  
Vol 108 (2) ◽  
pp. S74-S75
Author(s):  
Dawn Phillips ◽  
Lee Ann Lawson ◽  
Barbara Smith ◽  
Anatole Martin ◽  
Barry Byrne
Keyword(s):  
Replacement Therapy ◽  
Pompe Disease ◽  
Gene Replacement ◽  
Gene Replacement Therapy ◽  
Ventilatory Failure

Pre-clinical safety and efficacy findings of AT845, a novel gene replacement therapy for Pompe disease targeting skeletal muscle and heart

2020 ◽  
Vol 129 (2) ◽  
pp. S106
Author(s):  
Fulvio Mavilio ◽  
Justine Cunningham ◽  
Michelle Eggers ◽  
Garrett Heffner ◽  
Tim Stinchcombe ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Gene Replacement ◽  
Clinical Safety ◽  
Safety And Efficacy ◽  
Novel Gene ◽  
Gene Replacement Therapy

scholarly journals MEN1 Gene Replacement Therapy Reduces Proliferation Rates in a Mouse Model of Pituitary Adenomas

2012 ◽  
Vol 72 (19) ◽  
pp. 5060-5068 ◽  
Author(s):  
Gerard V. Walls ◽  
Manuel C. Lemos ◽  
Mahsa Javid ◽  
Miriam Bazan-Peregrino ◽  
Jeshmi Jeyabalan ◽  
...  
Keyword(s):  
Mouse Model ◽  
Replacement Therapy ◽  
Pituitary Adenomas ◽  
Gene Replacement ◽  
Men1 Gene ◽  
Gene Replacement Therapy

Delivery of cytosolic liver arginase into the mitochondrial matrix space: A possible novel site for gene replacement therapy

1996 ◽  
Vol 22 (6) ◽  
pp. 489-498 ◽  
Author(s):  
Paul B. Wissmann ◽  
Barbara K. Goodman ◽  
Joseph G. Vockley ◽  
Rita M. Kern ◽  
Stephen D. Cederbaum ◽  
...  
Keyword(s):  
Replacement Therapy ◽  
Gene Replacement ◽  
Mitochondrial Matrix ◽  
Matrix Space ◽  
Gene Replacement Therapy
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