Results 71 to 80 of about 11,459,182 (339)
Gene therapy for arthritis [PDF]
Modern Rheumatology, 2008 Arthritis is among the leading causes of disability in the developed world. There remains no cure for this disease and the current treatments are only modestly effective at slowing the disease's progression and providing symptomatic relief. The clinical effectiveness of current treatment regimens has been limited by short half-lives of the drugs and ...
Raphael Hirsch, Russell S. Traister
openaire +3 more sources
FV Vectors as Alternative Gene Vehicles for Gene Transfer in HSCs
Viruses, 2020 Hematopoietic Stem Cells (HSCs) are a unique population of cells, capable of reconstituting the blood system of an organism through orchestrated self-renewal and differentiation.
Emmanouil Simantirakis +2 more
doaj +1 more source
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 +10 more
doaj +1 more source
Single‐cell insights into the role of T cells in B‐cell malignancies
FEBS Letters, EarlyView.Single‐cell technologies have transformed our understanding of T cell–tumor cell interactions in B‐cell malignancies, revealing new T‐cell subsets, functional states, and immune evasion mechanisms. This Review synthesizes these findings, highlighting the roles of T cells in pathogenesis, progression, and therapy response, and underscoring their ...
Laura Llaó‐Cid
wiley +1 more source
Stem Cell Research & Therapy, 2016 Background Cirrhosis is a major health problem worldwide and new therapies are needed. Hepatic macrophages (hMø) have a pivotal role in liver fibrosis, being able to act in both its promotion and its resolution.
Esteban Fiore +10 more
doaj +1 more source
Gene Therapy for Hemoglobinopathies [PDF]
Human Gene Therapy, 2018 Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-globin gene under the transcriptional control of ...
Cavazzana M., Mavilio F.
openaire +3 more sources
Cyclic nucleotide signaling as a drug target in retinitis pigmentosa
FEBS Letters, EarlyView.Disruptions in cGMP and cAMP signaling can contribute to retinal dysfunction and photoreceptor loss in retinitis pigmentosa. This perspective examines the mechanisms and evaluates emerging evidence on targeting these pathways as a potential therapeutic strategy to slow or prevent retinal degeneration.
Katri Vainionpää +2 more
wiley +1 more source
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
BioDrugs, 2017 There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells ...
M. Naso +3 more
semanticscholar +1 more source
FEBS Letters, EarlyView.Exposure to common noxious agents (1), including allergens, pollutants, and micro‐nanoplastics, can cause epithelial barrier damage (2) in our body's protective linings. This may trigger an immune response to our microbiome (3). The epithelial barrier theory explains how this process can lead to chronic noncommunicable diseases (4) affecting organs ...
Can Zeyneloglu +17 more
wiley +1 more source