Results 51 to 60 of about 1,003,750 (315)
GENE THERAPY IN MUCOPOLYSACCHARIDOSIS TYPE IIIA: CASE REPORTS
Slovenska pediatrija, 2022 Mucopolysaccharidoses are a group of rare lysosomal storage diseases. The clinical signs develop gradually, the impairment is progressive and multiple organs are affected. With the currently known treatment options, the patient cannot be cured.
Benjamin Lah +7 more
doaj +1 more source
Pediatric Blood &Cancer, EarlyView.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
Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders
Molecular Therapy: Methods & Clinical Development, 2020 Many human genetic diseases are associated with gross mutations such as aneuploidies, deletions, duplications, or inversions. For these “structural” disorders, conventional gene therapy, based on viral vectors and/or on programmable nuclease-mediated ...
Marianna Paulis +12 more
doaj +1 more source
, 2013 Background & Aims: Genetic variations near the interferon lambda 3 gene (IFNL3, IL28B) are the most powerful predictors for sustained virologic response (SVR) in patients with chronic hepatitis C virus (HCV) infection, compared to other biochemical or ...
Simone Susser (48294) +41 more
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Wilms tumor (WT) treatment imposes a significant time burden on patients and their families. Time toxicity is a patient‐centered metric that quantifies the burden of healthcare interaction. We sought to define time toxicity in the first year after diagnosis of WT and hypothesized that it would increase as tumor stage and treatment ...
Caleb Q. Ashbrook +6 more
wiley +1 more source
Frontiers in Molecular Biosciences, 2019 Targeting cancer cells metabolism is promising strategy in inhibiting cancer cells progression that are known to exhibit increased aerobic glycolysis. We used the glucose analog 2-Deoxyglucose (2-DG) as a competitor molecule of glucose.
Ahmed Majeed Al-Shammari +3 more
doaj +1 more source
Pediatric Blood &Cancer, EarlyView.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
Mesenchymal and neural stem cell differentiation into Purkinje-like cells: Morphological study
Journal of Taibah University Medical Sciencesالملخص: أهداف البحث: تُعدّ خلايا بيركنجي فئةً محورية من الخلايا العصبية المُثبِّطة في الجهاز العصبي المركزي، وقد ارتبطخلل وظيفتها بطيفٍ من الاضطرابات العصبية.
Maeda H. Mohammad, PhD +4 more
doaj +1 more source
The Role of Hematopoietic Cell Transplantation in Ataxia‐Telangiectasia
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Ataxia‐telangiectasia (A‐T) is a DNA repair disorder characterized by neurodegeneration, immunodeficiency, and cancer predisposition. Hematopoietic cell transplantation (HCT) is an established therapy in related disorders such as Fanconi anemia (FA) and Nijmegen breakage syndrome (NBS), but its role in A‐T is unclear.
Laila Alkhouli +3 more
wiley +1 more source
BiologyNeural excitatory/inhibitory (E/I) imbalance plays a pivotal role in the aging process. However, despite its significant impact, the role of E/I imbalance in motor dysfunction and neurodegenerative diseases has not received sufficient attention.
Xuhui Chen +6 more
doaj +1 more source