Zobrazeno 1 - 3
of 3
pro vyhledávání: '"Amy Bastille"'
Autor:
Agnese Padula, Michele Spinelli, Edoardo Nusco, Xabier Bujanda Cundin, Filomena Capolongo, Severo Campione, Claudia Perna, Amy Bastille, Megan Ericson, Chih-Chieh Wang, Shengwen Zhang, Angela Amoresano, Mariana Nacht, Pasquale Piccolo
Publikováno v:
JCI Insight, Vol 8, Iss 21 (2023)
Application of classic liver-directed gene replacement strategies is limited in genetic diseases characterized by liver injury due to hepatocyte proliferation, resulting in decline of therapeutic transgene expression and potential genotoxic risk. Wil
Externí odkaz:
https://doaj.org/article/5d899573bc2c4dc9b7a0e014f3d372bf
Autor:
Shengwen Zhang, Amy Bastille, Susana Gordo, Nikhil Ramesh, Jenisha Vora, Elizabeth McCarthy, Xiaohan Zhang, Dylan Frank, Chih-Wei Ko, Carmen Wu, Noel Walsh, Shreya Amarwani, Jing Liao, Qiang Xiong, Lauren Drouin, Matthias Hebben, Kyle Chiang, B. Nelson Chau
Publikováno v:
PLoS ONE, Vol 17, Iss 9 (2022)
Methylmalonic acidemia (MMA) is an inborn error of metabolism mostly caused by mutations in the mitochondrial methylmalonyl-CoA mutase gene (MMUT). MMA patients suffer from frequent episodes of metabolic decompensation, which can be life threatening.
Externí odkaz:
https://doaj.org/article/5a7d34d7157b46f7b3fbfe75ccf6a783
Autor:
Shengwen Zhang, Amy Bastille, Susana Gordo, Nikhil Ramesh, Jenisha Vora, Elizabeth McCarthy, Xiaohan Zhang, Dylan Frank, Chih-Wei Ko, Carmen Wu, Noel Walsh, Shreya Amarwani, Jing Liao, Qiang Xiong, Lauren Drouin, Matthias Hebben, Kyle Chiang, B Nelson Chau
Publikováno v:
PLoS ONE, Vol 17, Iss 9, p e0274774 (2022)
Methylmalonic acidemia (MMA) is an inborn error of metabolism mostly caused by mutations in the mitochondrial methylmalonyl-CoA mutase gene (MMUT). MMA patients suffer from frequent episodes of metabolic decompensation, which can be life threatening.
Externí odkaz:
https://doaj.org/article/b1d5a26130fa4ac4b29586136934a317