Zobrazeno 1 - 10
of 23
pro vyhledávání: '"Giandomenico Turchiano"'
Autor:
Sriram Vaidyanathan, Jenny L. Kerschner, Alekh Paranjapye, Vrishti Sinha, Brian Lin, Tracy A. Bedrosian, Adrian J. Thrasher, Giandomenico Turchiano, Ann Harris, Matthew H. Porteus
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 1, Pp 102134- (2024)
A “universal strategy” replacing the full-length CFTR cDNA may treat >99% of people with cystic fibrosis (pwCF), regardless of their specific mutations. Cas9-based gene editing was used to insert the CFTR cDNA and a truncated CD19 (tCD19) enrichm
Externí odkaz:
https://doaj.org/article/b980cfbabc854f999ab24fb41f1df1a5
Autor:
Alessia Cavazza, Ayal Hendel, Rasmus O. Bak, Paula Rio, Marc Güell, Duško Lainšček, Virginia Arechavala-Gomeza, Ling Peng, Fatma Zehra Hapil, Joshua Harvey, Francisco G. Ortega, Coral Gonzalez-Martinez, Carsten W. Lederer, Kasper Mikkelsen, Giedrius Gasiunas, Nechama Kalter, Manuel A.F.V. Gonçalves, Julie Petersen, Alejandro Garanto, Lluis Montoliu, Marcello Maresca, Stefan E. Seemann, Jan Gorodkin, Loubna Mazini, Rosario Sanchez, Juan R. Rodriguez-Madoz, Noelia Maldonado-Pérez, Torella Laura, Michael Schmueck-Henneresse, Cristina Maccalli, Julian Grünewald, Gloria Carmona, Neli Kachamakova-Trojanowska, Annarita Miccio, Francisco Martin, Giandomenico Turchiano, Toni Cathomen, Yonglun Luo, Shengdar Q. Tsai, Karim Benabdellah
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 34, Iss , Pp 102066- (2023)
The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across co
Externí odkaz:
https://doaj.org/article/dff3b1dde38845db9319c7538193d46c
Autor:
Marta García, Jose Bonafont, Jesús Martínez-Palacios, Rudan Xu, Giandomenico Turchiano, Stina Svensson, Adrian J. Thrasher, Fernando Larcher, Marcela Del Rio, Rubén Hernández-Alcoceba, Marina I. Garín, Ángeles Mencía, Rodolfo Murillas
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 96-108 (2022)
Recessive dystrophic epidermolysis bullosa, a devastating skin fragility disease characterized by recurrent skin blistering, scarring, and a high risk of developing squamous cell carcinoma is caused by mutations in COL7A1, the gene encoding type VII
Externí odkaz:
https://doaj.org/article/d7c843aebbdf4538b32d4bed782a099b
Autor:
Rajeev Rai, Marianna Romito, Elizabeth Rivers, Giandomenico Turchiano, Georges Blattner, Winston Vetharoy, Dariusz Ladon, Geoffroy Andrieux, Fang Zhang, Marta Zinicola, Diego Leon-Rico, Giorgia Santilli, Adrian J. Thrasher, Alessia Cavazza
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-15 (2020)
In recent years, hematopoietic stem cells gene editing has emerged as a promising tool to treat blood disorders. Here the authors develop a CRISPR/Cas9-based genome editing strategy that allows the precise correction of Wiskott-Aldrich Syndrome in vi
Externí odkaz:
https://doaj.org/article/e93094c9c51f4c1aa746e611fd6ea64c
Publikováno v:
Frontiers in Genome Editing, Vol 2 (2020)
Gene editing technologies show great promise for application to human disease as a result of rapid developments in targeting tools notably based on ZFN, TALEN, and CRISPR-Cas systems. Precise modification of a DNA sequence is now possible in mature h
Externí odkaz:
https://doaj.org/article/2fcd0dcb4ee442fda5ffcae7a7d8ada3
Autor:
Giandomenico Turchiano, Maria Carmela Latella, Andreas Gogol Döring, Claudia Cattoglio, Fulvio Mavilio, Zsuzsanna Izsvák, Zoltán Ivics, Alessandra Recchia
Publikováno v:
PLoS ONE, Vol 15, Iss 1, p e0228703 (2020)
[This corrects the article DOI: 10.1371/journal.pone.0112712.].
Externí odkaz:
https://doaj.org/article/8cecaf2f5141488d85d844ad5386fc50
Autor:
Petros Patsali, Giandomenico Turchiano, Panayiota Papasavva, Marianna Romito, Constantinos C. Loucari, Coralea Stephanou, Soteroulla Christou, Maria Sitarou, Claudio Mussolino, Tatjana I. Cornu, Michael N. Antoniou, Carsten W. Lederer, Toni Cathomen, Marina Kleanthous
Publikováno v:
Haematologica, Vol 104, Iss 11 (2019)
Externí odkaz:
https://doaj.org/article/d0e06e164c7d47edb92e9f732efff970
Autor:
Giandomenico Turchiano, Maria Carmela Latella, Andreas Gogol-Döring, Claudia Cattoglio, Fulvio Mavilio, Zsuzsanna Izsvák, Zoltán Ivics, Alessandra Recchia
Publikováno v:
PLoS ONE, Vol 9, Iss 11, p e112712 (2014)
The Sleeping Beauty (SB) transposon is a non-viral integrating vector system with proven efficacy for gene transfer and functional genomics. However, integration efficiency is negatively affected by the length of the transposon. To optimize the SB tr
Externí odkaz:
https://doaj.org/article/eefaa821432f40aaad927477eac03a9c
Autor:
Georges Blattner, Fang Zhang, Winston Vetharoy, Marianna Romito, Adrian J. Thrasher, Elizabeth Rivers, Dariusz Ladon, Marta Zinicola, Diego Leon-Rico, Rajeev Rai, Giandomenico Turchiano, Giorgia Santilli, Geoffroy Andrieux, Alessia Cavazza
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-15 (2020)
Nature Communications
Nature Communications
Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency with severe platelet abnormalities and complex immunodeficiency. Although clinical gene therapy approaches using lentiviral vectors have produced encouraging results, full immune
Autor:
Valentina Pennucci, Julia Klermund, Giandomenico Turchiano, Claudio Mussolino, Sushmita Poddar, Toni Cathomen, Melanie Boerries, Tatjana I. Cornu, Melina el Gaz, Georges Blattner, Geoffroy Andrieux, Gianni Monaco
Publikováno v:
Cell stem cell. 28(6)
Genome editing has shown great promise for clinical translation but also revealed the risk of genotoxicity caused by off-target effects of programmable nucleases. Here we describe chromosomal aberrations analysis by single targeted linker-mediated PC