Zobrazeno 1 - 10
of 9 994
pro vyhledávání: '"lentiviral"'
Autor:
Keatdamrong Janpipatkul, Nareerat Sutjarit, Amornrat Tangprasittipap, Tai Chaiamarit, Pawarit Innachai, Kanoknetr Suksen, Tanida Chokpanuwat, Thipwimol Tim-Aroon, Usanarat Anurathapan, Natee Jearawiriyapaisarn, Alisa Tubsuwan, Supareak Bowornpinyo, Nithi Asavapanumas, Arthit Chairoungdua, Kanit Bhukhai, Suradej Hongeng
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-14 (2024)
Abstract Background Gaucher disease (GD) is one of the most common types of lysosomal storage diseases (LSDs) caused by pathogenic variants of lysosomal β-glucocerebrosidase gene (GBA1), resulting in the impairment of Glucocerebrosidase (GCase) enzy
Externí odkaz:
https://doaj.org/article/59a850ee25e242c784be1f2144fdcbd3
Autor:
Adrien Krug, Aymen Saidane, Chiara Martinello, Floriane Fusil, Alexander Michels, Christian J. Buchholz, Jean-Ehrland Ricci, Els Verhoeyen
Publikováno v:
Journal of Experimental & Clinical Cancer Research, Vol 43, Iss 1, Pp 1-18 (2024)
Abstract Background For angioimmunoblastic T cell lymphoma (AITL), a rare cancer, no specific treatments are available and survival outcome is poor. We previously developed a murine model for AITL that mimics closely human disease and allows to evalu
Externí odkaz:
https://doaj.org/article/daf8777c96c74d378e623fabf71e0eca
Autor:
Wuh-Liang Hwu
Publikováno v:
Journal of Biomedical Science, Vol 31, Iss 1, Pp 1-13 (2024)
Abstract Gene therapy has made considerable strides in recent years. More than 4000 protein-coding genes have been implicated in more than 6000 genetic diseases; next-generation sequencing has dramatically revolutionized the diagnosis of genetic dise
Externí odkaz:
https://doaj.org/article/c3989bfb13684df09009458ecb06391b
Autor:
Xueling Wu, Xiaoya Zhou, Yueming Wang, Jian Wu, Qian Liang, Xu Yang, Kehua Zhang, Shufang Meng
Publikováno v:
Heliyon, Vol 10, Iss 20, Pp e38512- (2024)
Lentiviruses, with their high transduction efficiency and gene expression levels, are widely used as gene delivery vectors in the development of chimeric antigen receptor T cells (CAR-T) and other genetically modified cell therapies. Accurate determi
Externí odkaz:
https://doaj.org/article/a9370f7fc62c4340b04bb2433cd26945
Autor:
Sara Penna, Alessandra Zecchillo, Martina Di Verniere, Elena Fontana, Valeria Iannello, Eleonora Palagano, Stefano Mantero, Andrea Cappelleri, Elena Rizzoli, Ludovica Santi, Laura Crisafulli, Marta Filibian, Antonella Forlino, Luca Basso-Ricci, Serena Scala, Eugenio Scanziani, Thorsten Schinke, Francesca Ficara, Cristina Sobacchi, Anna Villa, Valentina Capo
Publikováno v:
Frontiers in Endocrinology, Vol 15 (2024)
IntroductionAutosomal recessive osteopetrosis (ARO) is a rare genetic disease, characterized by increased bone density due to defective osteoclast function. Most of the cases are due to TCIRG1 gene mutation, leading to severe bone phenotype and death
Externí odkaz:
https://doaj.org/article/66acccbe99664eafa53503d6ac2fd709
Autor:
Michela Milani, Cesare Canepari, Simone Assanelli, Simone Merlin, Ester Borroni, Francesco Starinieri, Mauro Biffi, Fabio Russo, Anna Fabiano, Desirèe Zambroni, Andrea Annoni, Luigi Naldini, Antonia Follenzi, Alessio Cantore
Publikováno v:
EMBO Molecular Medicine, Vol 16, Iss 6, Pp 1427-1450 (2024)
Abstract Lentiviral vectors (LV) are efficient vehicles for in vivo gene delivery to the liver. LV integration into the chromatin of target cells ensures their transmission upon proliferation, thus allowing potentially life-long gene therapy followin
Externí odkaz:
https://doaj.org/article/b045f6dbf8624a8e8e0b19badc7c0b7b
Autor:
Arezoo Jamali, Naphang Ho, Angela Braun, Elham Adabi, Frederic B Thalheimer, Christian J Buchholz
Publikováno v:
EMBO Molecular Medicine, Vol 16, Iss 4, Pp 784-804 (2024)
Abstract Cytokine release syndrome (CRS) is a significant side-effect of conventional chimeric antigen receptor (CAR) T-cell therapy. To facilitate patient accessibility, short-term (st) CAR T cells, which are administered to patients only 24 h after
Externí odkaz:
https://doaj.org/article/3e9529b73a504bd2bc8f86fd1a55c755
Autor:
Ane Arrasate, Igone Bravo, Carlos Lopez-Robles, Ane Arbelaiz-Sarasola, Maddi Ugalde, Martha Lucia Meijueiro, Miren Zuazo, Ana Valero, Soledad Banos-Mateos, Juan Carlos Ramirez, Carmen Albo, Andrés Lamsfus-Calle, Marie J. Fertin
Publikováno v:
Biomedicines, Vol 12, Iss 10, p 2265 (2024)
Background/Objectives: To date, nearly 300 lentiviral-based gene therapy clinical trials have been conducted, with eight therapies receiving regulatory approval for commercialization. These advances, along with the increased number of advanced-phase
Externí odkaz:
https://doaj.org/article/1054e6cdf5bf4f15bcbfa59d5b63de34
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Autor:
Paul G. Ayoub, Julia Gensheimer, Lindsay Lathrop, Colin Juett, Jason Quintos, Kevin Tam, Jack Reid, Feiyang Ma, Curtis Tam, Grace E. McAuley, Devin Brown, Xiaomeng Wu, Ruixue Zhang, Kathryn Bradford, Roger P. Hollis, Gay M. Crooks, Donald B. Kohn
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 4, Pp 101323- (2024)
X-linked lymphoproliferative disease (XLP1) results from SH2D1A gene mutations affecting the SLAM-associated protein (SAP). A regulated lentiviral vector (LV), XLP-SMART LV, designed to express SAP at therapeutic levels in T, NK, and NKT cells, is cr
Externí odkaz:
https://doaj.org/article/af4d633eb4eb4c8ea8d67abff5e5fe26