Zobrazeno 1 - 10
of 4 710
pro vyhledávání: '"Lentiviral Vector"'
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:
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
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:
Ayoub, Paul G. 1, Gensheimer, Julia 3, 7, Lathrop, Lindsay 1, Juett, Colin 2, Quintos, Jason 2, Tam, Kevin 2, Reid, Jack 2, Ma, Feiyang 2, Tam, Curtis 2, McAuley, Grace E. 2, Brown, Devin 2, Wu, Xiaomeng 2, Zhang, Ruixue 2, Bradford, Kathryn 3, Hollis, Roger P. 2, Crooks, Gay M. 3, 4, 5, 6, 7, Kohn, Donald B. 1, 2, 3, 4, 5, ∗
Publikováno v:
In Molecular Therapy - Methods & Clinical Development 12 December 2024 32(4)
Autor:
Khare, Veenita 1, Cherqui, Stephanie 1, ∗
Publikováno v:
In Kidney International December 2024 106(6):1051-1061
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:
Loukia Touramanidou, Sonam Gurung, Claudiu A. Cozmescu, Dany Perocheau, Dale Moulding, Patrick F. Finn, Andrea Frassetto, Simon N. Waddington, Paul Gissen, Julien Baruteau
Publikováno v:
Cells, Vol 13, Iss 23, p 1979 (2024)
Recently approved adeno-associated viral (AAV) vectors for liver monogenic diseases haemophilia A and B are exemplifying the success of liver-directed viral gene therapy. In parallel, additional gene therapy strategies are rapidly emerging to overcom
Externí odkaz:
https://doaj.org/article/013dd91f64db4e359881d03982e919bc
Publikováno v:
Neurotherapeutics, Vol 21, Iss 4, Pp e00443- (2024)
Leukodystrophies are progressive single gene disorders affecting the white matter of the brain. Several gene therapy trials are in progress to address the urgent unmet need for this patient population. We performed a comprehensive literature review o
Externí odkaz:
https://doaj.org/article/b112d0cbfac741779d6130fcb3d448fc