Results 61 to 70 of about 11,317 (250)

A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies

open access: yesMolecular Therapy: Methods & Clinical Development, 2018
Reactivation of fetal hemoglobin (HbF) is a promising approach for the treatment of β-hemoglobinopathies and the targeting of genes involved in HbF regulation is under intensive investigation. Here, we established a nonhuman primate (NHP) transplantation
Olivier Humbert   +4 more
doaj   +1 more source

A novel, nondestructive, dried blood spot-based hematocrit prediction method using noncontact diffuse reflectance spectroscopy [PDF]

open access: yes, 2016
Dried blood spot (DBS) sampling is recognized as a valuable alternative sampling strategy both in research and in clinical routine. Although many advantages are associated with DBS sampling, its more widespread use is hampered by several issues, of which
Aalders, Maurice CG   +3 more
core   +4 more sources

Predictors of impending acute chest syndrome in patients with sickle cell anaemia. [PDF]

open access: yes, 2020
Acute chest syndrome (ACS) is a major complication of sickle cell anaemia (SCA) and a leading cause for hospital admissions and death. We aimed to study the spectrum of clinical and laboratory features of ACS and to assess the predisposing factors and ...
Al-Busaidi, Ikhlas   +5 more
core   +1 more source

Molecular genetics and pathophysiology of 17 beta-hydroxysteroid dehydrogenase 3 deficiency. [PDF]

open access: yes, 1996
Autosomal recessive mutations in the 17 beta-hydroxysteroid dehydrogenase 3 gene impair the formation of testosterone in the fetal testis and give rise to genetic males with female external genitalia.
Andersson, Stefan   +14 more
core   +1 more source

Late Onset Telomere Biology Disorder Presenting With Pancytopenia, Immune Dysregulation, Interstitial Lung Disease and Alopecia

open access: yes
American Journal of Hematology, EarlyView.
Bo A. Wan   +7 more
wiley   +1 more source

Circulating angiogenesis‐related biomarkers in sickle cell retinopathy and maculopathy

open access: yesBritish Journal of Haematology, EarlyView.
Summary Sickle cell disease (SCD), encompassing genotypes such as HbSS and HbSC, leads to retinal complications such as sickle cell retinopathy (SCR) and maculopathy (SCM) through poorly understood mechanisms. This study explored associations of a panel of circulating angiogenesis‐related factors with SCR and SCM.
Rajani P. Brandsen   +6 more
wiley   +1 more source

Dose‐dependent impairment of brain functional and microstructural connectivity during leukaemia chemotherapy

open access: yesBritish Journal of Haematology, EarlyView.
Prospective functional MRI brain and neurocognitive assessments were performed serially during intensive chemotherapy for a cohort of adults with newly diagnosed acute myeloid leukaemia to elucidate ‘chemobrain’ language phenomena. At completion of consolidation, significant reductions in functional and microstructural connectivity were observed ...
Ashleigh P. Scott   +17 more
wiley   +1 more source

Pandemic influenza A (H1N1v) infection in pediatric population: a multicenter study in a North-East area of Italy [PDF]

open access: yes, 2011
BACKGROUND: Data on clinical presentation, morbidity and mortality of 2009 pandemic influenza virus (H1N1v) in paediatric population are still emerging; most of the data so far available came from selected cohorts of children admitted to tertiary care ...
Liviana Da Dalt   +6 more
core   +2 more sources

The emerging role of non-coding RNAs in the pathogenesis of infantile hemangioma

open access: yesCancer Cell International
Infantile hemangioma (IH) is a common benign tumor that occurs in children, affecting both blood vessels and soft tissues. Its pathological features include abnormal proliferation of endothelial cells and an irregular vascular structure.
Najmaldin Saki   +3 more
doaj   +1 more source

Factors Related to Reduced Bone Density in β-Thalassemia Major Patients: Mazandaran Thalassemia Registry [PDF]

open access: yesMajallah-i Dānishgāh-i ’Ulūm-i Pizishkī-i Shahīd Ṣadūqī Yazd, 2023
Introduction: OsteoPenia and osteoPorosis are known to be one of the main comPlications of β-thalassemia major (β-TM). The aim of this study was to determine the factors associated with decreased bone density of these Patients.
Hossein Karami   +8 more
doaj  

Home - About - Disclaimer - Privacy