scholarly journals AAV9 gene replacement therapy for respiratory insufficiency in very‐long chain acyl‐CoA dehydrogenase deficiency

2019 ◽  
Vol 42 (5) ◽  
pp. 870-877 ◽  
Author(s):  
Marina Zieger ◽  
Allison M. Keeler ◽  
Terence R. Flotte ◽  
Mai K. ElMallah
Keyword(s):  
Replacement Therapy ◽  
Respiratory Insufficiency ◽  
Dehydrogenase Deficiency ◽  
Gene Replacement ◽  
Gene Replacement Therapy ◽  
Chain Acyl ◽  
Long Chain

Milder childhood form of very long-chain acyl-CoA dehydrogenase deficiency in a 6-year-old Japanese boy

2000 ◽  
Vol 159 (12) ◽  
pp. 908-911 ◽  
Author(s):  
Toshiaki Doi ◽  
Wataru Abo ◽  
Masaru Tateno ◽  
Kaori Hayashi ◽  
Tsukasa Hori ◽  
...  
Keyword(s):  
Dehydrogenase Deficiency ◽  
Chain Acyl ◽  
Old Japanese ◽  
Childhood Form ◽  
Long Chain

scholarly journals Rhabdomyolysis in the Military: Recognizing Late-Onset Very Long-Chain Acyl Co-A Dehydrogenase Deficiency

2006 ◽  
Vol 171 (7) ◽  
pp. 657-658 ◽  
Author(s):  
Jodi D. Hoffman ◽  
Robert D. Steiner ◽  
Lori Paradise ◽  
Carey O. Harding ◽  
Li Ding ◽  
...  
Keyword(s):  
Dehydrogenase Deficiency ◽  
Late Onset ◽  
Chain Acyl ◽  
The Military ◽  
Long Chain

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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