Zobrazeno 1 - 10 of 19 pro vyhledávání: '"Shilpita Sarcar"'
1
Akademický článek
Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering
Autor: Sara Martina Maffioletti, Shilpita Sarcar, Alexander B.H. Henderson, Ingra Mannhardt, Luca Pinton, Louise Anne Moyle, Heather Steele-Stallard, Ornella Cappellari, Kim E. Wells, Giulia Ferrari, Jamie S. Mitchell, Giulia E. Tyzack, Vassilios N. Kotiadis, Moustafa Khedr, Martina Ragazzi, Weixin Wang, Michael R. Duchen, Rickie Patani, Peter S. Zammit, Dominic J. Wells, Thomas Eschenhagen, Francesco Saverio Tedesco
Publikováno v: Cell Reports, Vol 23, Iss 3, Pp 899-908 (2018)
Summary: Generating human skeletal muscle models is instrumental for investigating muscle pathology and therapy. Here, we report the generation of three-dimensional (3D) artificial skeletal muscle tissue from human pluripotent stem cells, including i
Externí odkaz: https://doaj.org/article/8b22248a00a9498bb213b38f5d00c2e3
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2
Akademický článek
Modeling Skeletal Muscle Laminopathies Using Human Induced Pluripotent Stem Cells Carrying Pathogenic LMNA Mutations
Autor: Heather B. Steele-Stallard, Luca Pinton, Shilpita Sarcar, Tanel Ozdemir, Sara M. Maffioletti, Peter S. Zammit, Francesco Saverio Tedesco
Publikováno v: Frontiers in Physiology, Vol 9 (2018)
Laminopathies are a clinically heterogeneous group of disorders caused by mutations in LMNA. The main proteins encoded by LMNA are Lamin A and C, which together with Lamin B1 and B2, form the nuclear lamina: a mesh-like structure located underneath t
Externí odkaz: https://doaj.org/article/fa7575862e9f4e049bd373fa4c434189
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5
Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9
Autor: Marinee Chuah, Thierry VandenDriessche, Ermira Samara-Kuko, Nisha Nair, Kshitiz Singh, Shilpita Sarcar, Melvin Y Rincon, Hanneke Evens
Publikováno v: Molecular Therapy
In vivo tissue-specific genome editing at the desired loci is still a challenge. Here, we report that AAV9-delivery of truncated guide RNAs (gRNAs) and Cas9 under the control of a computationally designed hepatocyte-specific promoter lead to liver-sp
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::68bdd7332251bfd63a523f51d2b5bc38
https://doi.org/10.1016/j.ymthe.2018.02.023
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6
Next-generation muscle-directed gene therapy by in silico vector design
Autor: M Chuah, Maurilio Sampaolesi, H. Q. Pham, P. In't Veld, Mariana Loperfido, D. Boon, Melvin Y Rincon, Susan Jarmin, P.j. De Bleser, Ermira Samara-Kuko, Hanneke Evens, Emanuele Berardi, Jaitip Tipanee, Marleen Keyaerts, Thierry VandenDriessche, Shilpita Sarcar, George Dickson, Warut Tulalamba, Tony Lahoutte
Publikováno v: Nature Communications
NATURE COMMUNICATIONS
Nature Communications, Vol 10, Iss 1, Pp 1-16 (2019)
There is an urgent need to develop the next-generation vectors for gene therapy of muscle disorders, given the relatively modest advances in clinical trials. These vectors should express substantially higher levels of the therapeutic transgene, enabl
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2ec3527db24cdc10d4ced7f0039fd339
http://hdl.handle.net/11573/1581766
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8
Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy
Autor: Marleen Keyaerts, Shilpita Sarcar, Takis Athanasopoulos, Melvin Y Rincon, George Dickson, Ermira Samara-Kuko, Abel Acosta-Sanchez, Dina Danso-Abeam, Tony Lahoutte, Marinee Chuah, Thierry VandenDriessche, Janka Matrai, Pieter De Bleser
Publikováno v: Molecular Therapy
MOLECULAR THERAPY
Gene therapy is a promising emerging therapeutic modality for the treatment of cardiovascular diseases and hereditary diseases that afflict the heart. Hence, there is a need to develop robust cardiac-specific expression modules that allow for stable
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6f3a78691af44c22e537f158b9e3ab6c
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9
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy
Autor: Marinee Chuah, Beat Thöny, Omid Ghandeharian, Sumitava Dastidar, Thierry VandenDriessche, Pieter De Bleser, Nisha Nair, Hiu Man Viecelli, Emira Samara-Kuko, Hanneke Evens, Shilpita Sarcar, Melvin Y Rincon
Publikováno v: Blood. 123:3195-3199
The development of the next-generation gene therapy vectors for hemophilia requires using lower and thus potentially safer vector doses and augmenting their therapeutic efficacy. We have identified hepatocyte-specific transcriptional cis-regulatory m
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::83fc9e130b9de0700fd074b637f7a69d
https://doi.org/10.1182/blood-2013-10-534032
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10
Explant culture: a simple, reproducible, efficient and economic technique for isolation of mesenchymal stromal cells from human adipose tissue and lipoaspirate
Autor: Shilpita Sarcar, Anish Sen Majumdar, Nancy Priya, Swathi SundarRaj
Publikováno v: Journal of Tissue Engineering and Regenerative Medicine. 8:706-716
Adipose tissue has emerged as a preferred source of mesenchymal stem/stromal cells (MSC), due to its easy accessibility and high MSC content. The conventional method of isolation of adipose tissue-derived stromal cells (ASC) involves enzymatic digest
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::1786d17b81308089eb8b047995b7be4b
https://doi.org/10.1002/term.1569
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