Zobrazeno 1 - 10
of 21
pro vyhledávání: '"Mathieu Nonnenmacher"'
Autor:
Dirk Grimm, Mathieu Nonnenmacher
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 364-365 (2023)
Externí odkaz:
https://doaj.org/article/52ef8ed6413947b0a5838448080c3de2
Autor:
Mathieu Nonnenmacher, Wei Wang, Matthew A. Child, Xiao-Qin Ren, Carol Huang, Amy Zhen Ren, Jenna Tocci, Qingmin Chen, Kelsey Bittner, Katherine Tyson, Nilesh Pande, Charlotte Hiu-Yan Chung, Steven M. Paul, Jay Hou
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 366-378 (2021)
Therapeutic payload delivery to the central nervous system (CNS) remains a major challenge in gene therapy. Recent studies using function-driven evolution of adeno-associated virus (AAV) vectors have successfully identified engineered capsids with im
Externí odkaz:
https://doaj.org/article/fa7202f054c14b0abb47979c4151abb9
Autor:
Kleopatra Rapti, Francesca Stillitano, Ioannis Karakikes, Mathieu Nonnenmacher, Thomas Weber, Jean-Sebastian Hulot, Roger J Hajjar
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss , Pp - (2015)
Human embryonic stem cells (hESC) and induced pluripotent stem cells (hiPSC) assert a great future for the cardiovascular diseases, both to study them and to explore therapies. However, a comprehensive assessment of the viral vectors used to modify t
Externí odkaz:
https://doaj.org/article/298f46d9a0274776840904672ef93bd8
Autor:
Jaydev Dave, Nour Raad, Nishka Mittal, Lu Zhang, Anthony Fargnoli, Jae Gyun Oh, Maria Elisabetta Savoia, Jens Hansen, Marika Fava, Xiaoke Yin, Konstantinos Theofilatos, Delaine Ceholski, Erik Kohlbrenner, Dongtak Jeong, Lauren Wills, Mathieu Nonnenmacher, Kobra Haghighi, Kevin D Costa, Irene C Turnbull, Manuel Mayr, Chen-Leng Cai, Evangelia G Kranias, Fadi G Akar, Roger J Hajjar, Francesca Stillitano
Publikováno v:
Cardiovasc Res
Aims A mutation in the phospholamban (PLN) gene, leading to deletion of Arg14 (R14del), has been associated with malignant arrhythmias and ventricular dilation. Identifying pre-symptomatic carriers with vulnerable myocardium is crucial because arrhyt
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5dbedb98cc0fa6633afea4ced1ed20e3
https://doi.org/10.1093/cvr/cvac021
https://doi.org/10.1093/cvr/cvac021
Autor:
Daniel G. Anderson, Kenneth Fish, Irene C. Turnbull, Jiqiu Chen, Mathieu Nonnenmacher, Roger J. Hajjar, Kevin D. Costa, Kiyotake Ishikawa, Ahmed A. Eltoukhy
Publikováno v:
Molecular Therapy. 24:66-75
Nanoparticle-based delivery of nucleotides offers an alternative to viral vectors for gene therapy. We report highly efficient in vivo delivery of modified mRNA (modRNA) to rat and pig myocardium using formulated lipidoid nanoparticles (FLNP). Direct
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b146ab1ae029a3cce1ff1533342bdeb8
https://doi.org/10.1038/mt.2015.193
https://doi.org/10.1038/mt.2015.193
Autor:
Ninette Cohen, Chi Wing Kong, Joshua Mayourian, Razvan L. Cornea, Johan L.M. Björkegren, Ronald A. Li, Irene C. Turnbull, Angelos A. Skodras, Kevin D. Costa, Przemek A. Gorski, Daniel R. Stroik, David D. Thomas, Simon Koplev, Delaine K. Ceholski, Mathieu Nonnenmacher, Roger J. Hajjar, Francesca Stillitano
Dilated cardiomyopathy (DCM) can be caused by mutations in the cardiac protein phospholamban (PLN). We used CRISPR/Cas9 to insert the R9C PLN mutation at its endogenous locus into a human induced pluripotent stem cell (hiPSC) line from an individual
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2bae39d7e3858f0041582f46225398c9
https://europepmc.org/articles/PMC6039110/
https://europepmc.org/articles/PMC6039110/
Autor:
Mathieu Nonnenmacher, Jean-Sébastien Hulot, Roger J. Hajjar, Francesca Stillitano, Evangelia G. Kranias, Irene C. Turnbull, Kevin D. Costa, Peter Backeris, Ioannis Karakikes
Publikováno v:
European Heart Journal. 37:3282-3284
In this study, we used three-dimensional human engineered cardiac tissue technology to directly show that phospholamban (PLN) R14del mutation impairs cardiac contractility and to demonstrate restoration of contractile properties with targeted genetic
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::693fdb8389db760548431c545b095879
https://doi.org/10.1093/eurheartj/ehw307
https://doi.org/10.1093/eurheartj/ehw307
Autor:
Delaine K Ceholski, Irene C Turnbull, Simon Koplev, Chi-Wing Kong, Francesca Stillitano, Mathieu Nonnenmacher, Kobra Haghighi, Przemyslaw A Gorski, Ninette Cohen, Fotis Kolokathis, Dimitrios T Kremastinos, Arvind Babu, Ronald A Li, Chiara Giannarelli, Ioannis Karakikes, Evangelia G Kranias, Kevin D Costa, Roger J Hajjar
Publikováno v:
Circulation Research. 121
Dilated cardiomyopathy (DCM) can be caused by genetic mutations in numerous cardiac proteins, including phospholamban (PLN). PLN mutations are quite rare and obtaining patient samples for mechanistic insights can be challenging. We used genome editin
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::793b0f7a957d6b6391660e6d308e4d33
https://doi.org/10.1161/res.121.suppl_1.185
https://doi.org/10.1161/res.121.suppl_1.185
Autor:
Dongzhu Jin, Thomas Weber, Jason C. Kovacic, Roger J. Hajjar, Fadi G. Akar, Erik Kohlbrenner, Antoine H. Chaanine, Mathieu Nonnenmacher
Publikováno v:
Gene therapy
Adeno-associated virus (AAV)-based vectors are promising vehicles for therapeutic gene delivery, including for the treatment for heart failure. It has been demonstrated for each of the AAV serotypes 1 through 8 that inhibition of the proteasome resul
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ad3e5b109bbac3fe05baeee7768f3f4b
http://europepmc.org/articles/PMC3976435
http://europepmc.org/articles/PMC3976435
Autor:
Changwon Kho, Daniel S Matasic, Roger J. Hajjar, Jean-Sébastien Hulot, Ludovic Benard, Mathieu Nonnenmacher, Erik Kohlbrenner, Ahyoung Lee, Catherine Pavoine, Marine Cacheux, Jae Gyun Oh
Background— Stromal interaction molecule 1 (STIM1) is a dynamic calcium signal transducer implicated in hypertrophic growth of cardiomyocytes. STIM1 is thought to act as an initiator of cardiac hypertrophic response at the level of the sarcolemma,
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::259ab66bc4f9306be12c7e4df9fd3dc2
https://europepmc.org/articles/PMC4829441/
https://europepmc.org/articles/PMC4829441/