Zobrazeno 1 - 10
of 16
pro vyhledávání: '"Hiu Man Grisch-Chan"'
Autor:
Sereina Deplazes, Andrea Schlegel, Zhuolun Song, Gabriella Allegri, Nicole Rimann, Tanja Scherer, Melanie Willimann, Lennart Opitz, Sharon C. Cunningham, Ian E. Alexander, Anja Kipar, Johannes Häberle, Beat Thöny, Hiu Man Grisch-Chan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 352-367 (2022)
Hydrodynamic tail vein injection (HTV) is the “gold standard” for delivering naked DNA vectors to mouse liver, thereby transfecting predominately perivenous hepatocytes. While HTV corrects metabolic liver defects such as phenylketonuria or cystat
Externí odkaz:
https://doaj.org/article/334928f0d4ad4afba5bcb442c5af482b
Autor:
Tatjana Chan, Hiu Man Grisch-Chan, Philipp Schmierer, Ulrike Subotic, Nicole Rimann, Tanja Scherer, Udo Hetzel, Matthias Bozza, Richard Harbottle, James A. Williams, Barbara Steblaj, Simone K. Ringer, Johannes Häberle, Xaver Sidler, Beat Thöny
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 268-279 (2022)
Hepatic gene therapy by delivering non-integrating therapeutic vectors in newborns remains challenging due to the risk of dilution and loss of efficacy in the growing liver. Previously we reported on hepatocyte transfection in piglets by intraportal
Externí odkaz:
https://doaj.org/article/9af6b8771dc44a3caf3414a328b73cff
Autor:
Jonas Buck, Dennis Mueller, Ute Mettal, Miriam Ackermann, Hiu Man Grisch-Chan, Beat Thöny, Andreas Zumbuehl, Jörg Huwyler, Dominik Witzigmann
Publikováno v:
ACS Omega, Vol 5, Iss 38, Pp 24724-24732 (2020)
Externí odkaz:
https://doaj.org/article/78406cbe61a24233baa11011570fcf0f
Autor:
Hiu Man Grisch-Chan, Andrea Schlegel, Tanja Scherer, Gabriella Allegri, Raphael Heidelberger, Panagiota Tsikrika, Marco Schmeer, Martin Schleef, Cary O. Harding, Johannes Häberle, Beat Thöny
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 7, Iss C, Pp 339-349 (2017)
Limited duration of transgene expression, insertional mutagenesis, and size limitations for transgene cassettes pose challenges and risk factors for many gene therapy vectors. Here, we report on physiological expression of liver phenylalanine hydroxy
Externí odkaz:
https://doaj.org/article/6d2ec87b2b7140dbbef77951976665fb
Autor:
Tanja Rothgangl, Martina Hruzova, Ralph Gnannt, Nadja Aeberhard, Lucas Kissling, Hiu Man Grisch-Chan, Sven Klassa, Nicole Rimann, Kim F. Marquart, Eleonora Ioannidi, Anja Wolf, Christian Kupatt, Xaver Sidler, Johannes Häberle, Gerald Schwank, Beat Thöny
One challenge for liver-directed gene therapy is sufficient vector delivery to the target tissue while minimizing loss of the applied vector dose to other tissues. Infusion via peripheral veins is the least invasive approach; however, it results in s
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::16e29a86e47747a7ab0e74f492719f8e
https://doi.org/10.1101/2023.04.05.535660
https://doi.org/10.1101/2023.04.05.535660
Autor:
Jens Casper, Laura Nicolle, Melanie Willimann, Evrim Ümit Kuzucu, Alan Tran, Perrine Robin, Pascal Detampel, Hiu Man Grisch‐Chan, Beat Thöny, Jörg Huwyler, Sandrine Gerber‐Lemaire
Publikováno v:
Macromolecular bioscience.
The delivery of nucleic acids relies on vectors that condense and encapsulate their cargo. Especially nonviral gene delivery systems are of increasing interest. However, low transgene expression levels and limited tolerability of these systems remain
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7f00119083522d323d5ccbbc056eb8a3
https://pubmed.ncbi.nlm.nih.gov/36200651
https://pubmed.ncbi.nlm.nih.gov/36200651
Autor:
Desirée Böck, Tanja Rothgangl, Lukas Villiger, Lukas Schmidheini, Mai Matsushita, Nicolas Mathis, Eleonora Ioannidi, Nicole Rimann, Hiu Man Grisch-Chan, Susanne Kreutzer, Zacharias Kontarakis, Manfred Kopf, Beat Thöny, Gerald Schwank
Publikováno v:
Science Translational Medicine, 14 (636)
Sci Transl Med
Sci Transl Med
Prime editing is a highly versatile CRISPR-based genome editing technology that works without DNA double-strand break formation. Despite rapid technological advances, in vivo application for the treatment of genetic diseases remains challenging. Here
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a1da162c702a5cff0210cef62d5407b0
https://www.zora.uzh.ch/id/eprint/219573/
https://www.zora.uzh.ch/id/eprint/219573/
Publikováno v:
Hum Gene Ther
Phenylketonuria (PKU) is considered to be a paradigm for a monogenic metabolic disorder but was never thought to be a primary application for human gene therapy due to established alternative treatment. However, somewhat unanticipated improvement in
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9572e87174b22af13b083c51e3d45e58
https://doi.org/10.1089/hum.2019.111
https://doi.org/10.1089/hum.2019.111
Autor:
Lorena Gallego-Villar, Beat Thöny, Hiu Man Grisch-Chan, Warren D. Kruger, Johannes Häberle, Hyung-Ok Lee
Publikováno v:
Hum Gene Ther
Cystathionine β-synthase (CBS) deficiency is a recessive inborn error of metabolism characterized by extremely elevated total homocysteine (tHcy) in the blood. Patients diagnosed with CBS deficiency have a variety of clinical problems, including dis
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4f963e75ce0431f597c699a24a875689
https://doi.org/10.1089/hum.2019.014
https://doi.org/10.1089/hum.2019.014
Autor:
Anna Klimovskaia, Beat Thöny, Ralph Fingerhut, Jakub Krijt, Jean-Marc Nuoffer, Gabriella Allegri, Hiu Man Grisch-Chan, Viktor Kožich, Benjamin Causton, Tanja Scherer, Johannes Häberle, Sereina Deplazes, Carmen Diez-Fernandez, Nicole Rimann, Jonathan W. Leff
Publikováno v:
Journal of Inherited Metabolic Disease. 42:1064-1076
The most common ureagenesis defect is X-linked ornithine transcarbamylase (OTC) deficiency which is a main target for novel therapeutic interventions. The spf ash mouse model carries a variant (c.386G>A, p.Arg129His) that is also found in patients. M
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::bf3e0a821a69a301885b0f46378e2962
https://doi.org/10.1002/jimd.12068
https://doi.org/10.1002/jimd.12068