Results 61 to 70 of about 2,093,614 (359)

Clinical Characteristics and Prognostic Risk Factors for Pediatric B‐Cell Lymphoblastic Lymphoma: A Multicenter Retrospective Cohort Study for China Net Childhood Lymphoma

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background B‐cell lymphoblastic lymphoma (B‐LBL) represents a rare variety of non‐Hodgkin lymphoma, with limited research on its biology, progression, and management. Methods A retrospective analysis was performed on the clinical characteristics of 256 patients aged ≤18 years who received treatment under the China Net Childhood Lymphoma (CNCL)‐
Zhijuan Liu, Jian Wang, Ling Jin, Yanlong Duan, Fu Li, Yueping Jia, Leping Zhang, Xiaojun Yuan, Wei Liu, Ying Liu, Ansheng Liu, Mincui Zheng, Yunpeng Dai, Lian Jiang, Lirong Sun, Xiaowen Zhai, Rong Liu, Chunju Zhou, Zifen Gao, Yonghong Zhang, Xiaomei Yang   +20 more
wiley   +1 more source

FGF21 gene therapy as treatment for obesity and insulin resistance

EMBO Molecular Medicine, 2018
Prevalence of type 2 diabetes (T2D) and obesity is increasing worldwide. Currently available therapies are not suited for all patients in the heterogeneous obese/T2D population, hence the need for novel treatments.
Veronica Jimenez, Claudia Jambrina, Estefania Casana, Victor Sacristan, Sergio Muñoz, Sara Darriba, Jordi Rodó, Cristina Mallol, Miquel Garcia, Xavier León, Sara Marcó, Albert Ribera, Ivet Elias, Alba Casellas, Ignasi Grass, Gemma Elias, Tura Ferré, Sandra Motas, Sylvie Franckhauser, Francisca Mulero, Marc Navarro, Virginia Haurigot, Jesus Ruberte, Fatima Bosch   +23 more
doaj   +1 more source

Aurintricarboxylic acid increases yield of HSV-1 vectors

Molecular Therapy: Methods & Clinical Development, 2014
Production of large quantities of viral vectors is crucial for the success of gene therapy in the clinic. There is a need for higher titers of herpes simplex virus-1 (HSV-1) vectors both for therapeutic use as well as in the manufacturing of clinical ...
Peter Pechan, Jeffery Ardinger, Jyothi Ketavarapu, Hillard Rubin, Samuel C Wadsworth, Abraham Scaria   +5 more
doaj   +1 more source

ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing

BMC Genomics, 2020
Background Identifying nuclease-induced double-stranded breaks in DNA on a genome-wide scale is critical for assessing the safety and efficacy of genome editing therapies.
Camilo Breton, Peter M. Clark, Lili Wang, Jenny A. Greig, James M. Wilson   +4 more
doaj   +1 more source

Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression

Scientific Reports, 2022
High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials.
Anna V. Starikova, Victoria V. Skopenkova, Anna V. Polikarpova, Denis A. Reshetov, Svetlana G. Vassilieva, Oleg A. Velyaev, Anna A. Shmidt, Irina M. Savchenko, Vladislav O. Soldatov, Tatiana V. Egorova, Maryana V. Bardina   +10 more
doaj   +1 more source

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. [PDF]

, 2013
Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness.
Boye, S, Boye, SE, Chiodo, V, Dyka, F, Fofo, H, Hauswirth, WW, Lopes, VS, Louie, CM, Williams, DS   +8 more
core   +1 more source

Enteropathogenic E. coli shows delayed attachment and host response in human jejunum organoid‐derived monolayers compared to HeLa cells

FEBS Letters, EarlyView.
Enteropathogenic E. coli (EPEC) infects the human intestinal epithelium, resulting in severe illness and diarrhoea. In this study, we compared the infection of cancer‐derived cell lines with human organoid‐derived models of the small intestine. We observed a delayed in attachment, inflammation and cell death on primary cells, indicating that host ...
Mastura Neyazi, Pilar Samperio Ventayol, Natalie Burkard, Nicolas Schlegel, Carmen Aguilar, Sina Bartfeld   +5 more
wiley   +1 more source

Gene Therapy [PDF]

Internal Medicine, 1993
F, Levine, T, Friedmann
  +8 more sources

Early induction of cytokine release syndrome by rapidly generated CAR T cells in preclinical models

EMBO Molecular Medicine
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 vector ...
Arezoo Jamali, Naphang Ho, Angela Braun, Elham Adabi, Frederic B Thalheimer, Christian J Buchholz   +5 more
doaj   +1 more source

Expanding CAR T cells in human platelet lysate renders T cells with in vivo longevity

Journal for ImmunoTherapy of Cancer, 2019
Background Pre-clinical and clinical studies have shown that the infusion of CAR T cells with a naive-like (TN) and central memory (TCM) phenotype is associated with prolonged in vivo T cell persistence and superior anti-tumor effects.
Alejandro Torres Chavez, Mary Kathryn McKenna, Emanuele Canestrari, Christina T. Dann, Carlos A. Ramos, Premal Lulla, Ann M. Leen, Juan F. Vera, Norihiro Watanabe   +8 more
doaj   +1 more source