Results 61 to 70 of about 1,824,389 (266)
Background Acute lung injury (ALI) following pneumonia involves uncontrolled inflammation and tissue injury, leading to high mortality. We previously confirmed the significantly increased cargo content and extracellular vesicle (EV) production in ...
Yuna Bang+8 more
doaj +1 more source
Aurintricarboxylic acid increases yield of HSV-1 vectors
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+5 more
doaj +1 more source
Mesenchymal stem cell therapy for liver disease: full of chances and challenges
Liver disease is a major health problem that endangers human health worldwide. Currently, whole organ allograft transplantation is the gold standard for the treatment of end-stage liver disease.
Xue Yang+5 more
doaj +1 more source
Gene Therapy in Cardiac Arrhythmias [PDF]
Gene therapy has progressed from a dream to a bedside reality in quite a few human diseases. From its first application in adenosine deaminase deficiency, through the years, its application has evolved to vascular angiogenesis and cardiac arrhythmias.
Francis, Johnson+2 more
core
Clinical and biochemical response to neridronate treatment in a patient with osteoporosis-pseudoglioma syndrome (OPPG) [PDF]
Osteoporosis-pseudoglioma syndrome (OPPG) is a rare autosomal recessive syndrome characterized by juvenile-onset osteoporosis and ocular abnormalities due to a low-density lipoprotein receptor-related protein 5 (LRP5) gene mutation.
Celli, L+7 more
core +1 more source
Expanding CAR T cells in human platelet lysate renders T cells with in vivo longevity
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+8 more
doaj +1 more source
This book aims at providing an up-to-date report to cover key aspects of existing problems in the emerging field of targets in gene therapy. With the contributions in various disciplines of gene therapy, the book brings together major approaches: Target ...
core +1 more source
Gene therapy for hemophilia [PDF]
Hemophilia A and B are X-linked monogenic disorders resulting from deficiencies of factor VIII and FIX, respectively. Purified clotting factor concentrates are currently intravenously administered to treat hemophilia, but this treatment is non-curative.
Thierry VandenDriessche+3 more
openaire +3 more sources
FGF21 gene therapy as treatment for obesity and insulin resistance
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+23 more
doaj +1 more source
Autoimmune dysregulation and purine metabolism in adenosine deaminase (ADA)-deficiency
Genetic defects in the adenosine deaminase (ADA) gene are among the most common causes for severe combined immunodeficiency (SCID). ADA-SCID patients suffer from lymphopenia, severely impaired cellular and humoral immunity, failure to thrive and ...
Aisha Vanessa Sauer+5 more
doaj +1 more source