Zobrazeno 1 - 5
of 5
pro vyhledávání: '"Claudia Milazzo"'
Autor:
Martin H. Kang, Laura P. van Lieshout, Liqun Xu, Jakob M. Domm, Arul Vadivel, Laurent Renesme, Christian Mühlfeld, Maria Hurskainen, Ivana Mižíková, Yanlong Pei, Jacob P. van Vloten, Sylvia P. Thomas, Claudia Milazzo, Chanèle Cyr-Depauw, Jeffrey A. Whitsett, Lawrence M. Nogee, Sarah K. Wootton, Bernard Thébaud
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-20 (2020)
Surfactant protein B (SP-B) deficiency is a genetic lung disease that results in lethal respiratory distress within months of birth. Here, the authors describe a gene therapy strategy using a rationally designed AAV6 capsid that restores surfactant h
Externí odkaz:
https://doaj.org/article/6438f94f53b5451b8361dfa466593f2a
Autor:
Claudia Milazzo, Edwin J. Mientjes, Ilse Wallaard, Søren Vestergaard Rasmussen, Kamille Dumong Erichsen, Tejaswini Kakunuri, A.S. Elise van der Sman, Thomas Kremer, Meghan T. Miller, Marius C. Hoener, Ype Elgersma
Publikováno v:
JCI Insight, Vol 6, Iss 15 (2021)
Angelman syndrome (AS) is a severe neurodevelopmental disorder for which only symptomatic treatment with limited benefits is available. AS is caused by mutations affecting the maternally inherited ubiquitin protein ligase E3A (UBE3A) gene. Previous s
Externí odkaz:
https://doaj.org/article/4d87e9763e67445f825996968575d00d
Autor:
Judith C. Birkhoff, Anne L. Korporaal, Rutger W. W. Brouwer, Karol Nowosad, Claudia Milazzo, Lidia Mouratidou, Mirjam C. G. N. van den Hout, Wilfred F. J. van IJcken, Danny Huylebroeck, Andrea Conidi
Publikováno v:
Genes
Volume 14
Issue 3
Pages: 629
Volume 14
Issue 3
Pages: 629
Perturbation and mechanistic studies have shown that the DNA-binding transcription factor Zeb2 controls cell fate decision, differentiation and/or maturation in multiple cell lineages in embryos and after birth. In cultured embryonic stem cells (ESCs
Autor:
Rasmussen Søren Vestergaard, A S Elise van der Sman, Marius C. Hoener, Ype Elgersma, Claudia Milazzo, Tejaswini Kakunuri, Ilse Wallaard, Edwin Mientjes, Kamille Dumong Erichsen, Thomas Kremer, Meghan T. Miller
Publikováno v:
JCI Insight
JCI insight, 6(15):e145991. The American Society for Clinical Investigation
JCI insight, 6(15):e145991. The American Society for Clinical Investigation
Angelman syndrome (AS) is a severe neurodevelopmental disorder for which only symptomatic treatment with limited benefits is available. AS is caused by mutations affecting the maternally inherited ubiquitin protein ligase E3A (UBE3A) gene. Previous s
Autor:
Laurent Renesme, Liqun Xu, Sylvia P. Thomas, Christian Mühlfeld, Yanlong Pei, Bernard Thébaud, Chanèle Cyr-Depauw, Jeffrey A. Whitsett, Arul Vadivel, Lawrence M. Nogee, Ivana Mižíková, Maria Hurskainen, Claudia Milazzo, Laura P. van Lieshout, Martin H. Kang, Sarah K. Wootton, Jacob P. van Vloten, Jakob M. Domm
Publikováno v:
Nature Communications
Nature Communications, Vol 11, Iss 1, Pp 1-20 (2020)
Nature Communications, Vol 11, Iss 1, Pp 1-20 (2020)
Surfactant protein B (SP-B) deficiency is an autosomal recessive disorder that impairs surfactant homeostasis and manifests as lethal respiratory distress. A compelling argument exists for gene therapy to treat this disease, as de novo protein synthe