Results 41 to 50 of about 2,117,768 (268)
Optimized riboswitch-regulated AAV vector for VEGF-B gene therapy
Gene therapy would greatly benefit from a method to regulate therapeutic gene expression temporally. Riboswitches are small RNA elements that have been studied for their potential use in turning transgene expression on or off by ligand binding.
Reetta A. E. Eriksson +16 more
doaj +1 more source
The Systematic Production of Cells for Cell Therapies [PDF]
Stem cells have emerged as the starting material of choice for bioprocesses to produce cells and tissues to treat degenerative, genetic, and immunological disease. Translating the biological properties and potential of stem cells into therapies will require overcoming significant cell-manufacturing and regulatory challenges.
Kirouac, Daniel C., Zandstra, Peter W.
openaire +2 more sources
Abstract Systemic lupus erythematosus (SLE) is a chronic autoimmune and inflammatory disease with multiple organs and systems involved such as the kidney, lung, brain and the hematopoietic system. Although increased knowledge of the disease pathogenesis has improved treatment options, current immunosuppressive therapies have failed to ...
Yuan, Xinran, Sun, Lingyun
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ABSTRACT Background An internal tandem duplication in the gene encoding Fms‐like tyrosine kinase 3 (FLT3‐ITD) is associated with high relapse risk and poor prognosis in acute myeloid leukemia (AML) and plays a crucial role in treatment decisions. Measurable residual disease (MRD) analysis of FLT3‐ITD during and after treatment has shown prognostic ...
Sofie Johansson Alm +11 more
wiley +1 more source
Human induced pluripotent stem cells (iPSC) have demonstrated massive potentials for use in regenerative and personalized medicine due to their ability to expand in culture and differentiate into specialized cells with therapeutic benefits.
Sahana Suresh Babu +8 more
doaj +1 more source
ABSTRACT Claudin‐6 has emerged as a promising immunotherapeutic target, yet protein‐level data in atypical teratoid/rhabdoid tumors (AT/RTs) have been inconsistent. We analyzed 36 well‐characterized AT/RT samples and found membranous claudin‐6 protein expression in 58% of cases, with striking enrichment in the molecular subgroup AT/RT‐TYR (100%) and ...
Victoria E. Fincke +4 more
wiley +1 more source
ABSTRACT Background Central nervous system (CNS) involvement in childhood acute lymphoblastic leukemia (ALL) is assessed by cell counting and cytomorphology from cerebrospinal fluid (CSF) and is used for treatment stratification worldwide. The ratio of “CNS2” patients in clinical trials ranges from 3% to 40%, with unclear prognostic significance ...
Laura Almási +14 more
wiley +1 more source
ABSTRACT Immune effector cell‐associated hemophagocytic lymphohistiocytosis‐like syndrome (IEC‐HS) is a life‐threatening hyperinflammatory toxicity distinct from cytokine release syndrome (CRS) and neurotoxicity following chimeric antigen receptor T‐cell (CAR‐T) therapy. In a single‐institution retrospective cohort of pediatric and young adult patients
Thomas J. Galletta +6 more
wiley +1 more source
CAR-based cell therapies have shown clinical success in treating various cancers, with CAR T cell therapies entering the clinical route and CAR NK cell therapies being evaluated in early-stage clinical trials.
Katharina Schindler-Wnek +5 more
doaj +1 more source
Adipose mesenchymal stem cells (ASC) are considered minimally immunogenic. This is due to the low expression of human leukocyte antigens I (HLA-I), lack of HLA-II expression and low expression of co-stimulatory molecules such as CD40 and CD80.
Alvaro Avivar-Valderas +14 more
doaj +1 more source

