Results 151 to 160 of about 1,827,024 (396)

Antiviral response and HIV-1 inhibition in sickle cell disease

open access: yesiScience
Summary: Sickle cell disease (SCD) is characterized by hemolysis, vaso-occlusion, and ischemia. HIV-1 infection was previously shown to be suppressed in SCD PBMCs.
Namita Kumari   +10 more
doaj  

Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency [PDF]

open access: yes, 2018
Purpose: Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining ...
Ionova, Tatyana   +3 more
core   +1 more source

Prenatal Exome Sequencing Identifies Dual Maternal‐Fetal Diagnosis of HbF Mission Bay, a Novel HBG2 Variant Associated With Methemoglobinemia, Hypoxia and Hemolytic Anemia

open access: yesPrenatal Diagnosis, EarlyView.
ABSTRACT Prenatal exome sequencing (ES) can establish rare genetic diagnoses in a fetus but may also lead to occult genetic diagnosis in a biological parent. We present a case of dual fetal and maternal diagnosis by prenatal ES, in a fetus with unexplained anemia and in a pregnant patient with sickle cell disease (SCD) and recurrent unexplained hypoxia.
Matthew A. Shear   +6 more
wiley   +1 more source

Head shape determination and cranial evaluation using anatomical landmarks in children with sickle cell disease (SCD)

open access: yesAsian Journal of Medical Sciences, 2014
Background: Sickle cell disease is one of the commonest diseases widespread in sub-Saharan Africa and Nigeria in particular. This study investigated the possible effects of sickle cell disease (SCD) on the cranial index and pattern of head shapes of ...
MA Musa
doaj   +1 more source

Coexisting Cardiac and Hematologic Disorders. [PDF]

open access: yes, 2016
Patients with concomitant cardiac and hematologic disorders presenting for noncardiac surgery are challenging. Anemic patients with cardiac disease should be approached in a methodical fashion.
Goldhammer, Jordan E., Kohl, Benjamin A.
core   +3 more sources

Emerging disease-modifying therapies for sickle cell disease

open access: yesHaematologica, 2019
Sickle cell disease afflicts millions of people worldwide and approximately 100,000 Americans. Complications are myriad and arise as a result of complex pathological pathways ‘downstream’ to a point mutation in DNA, and include red blood cell membrane ...
Marcus A. Carden, J. Little
semanticscholar   +1 more source

Sickle Cell Disease Complications [PDF]

open access: yesThalassemia Reports, 2014
Sickle cell disease (SCD) is an inherited, lifelong condition. The sickle mutation consists a single nucleotide change (GAT->GTT) in the sixth codon of exon 1 of the β-globin gene coding for the β-globin polypeptide of hemoglobin (Hb) (a2β2). This change results in replacement of the wild type glutamic acid residue by a valine residue in β-globin ...
openaire   +3 more sources

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

open access: yesSmall Methods, EarlyView.
The review presents a comprehensive overview of each component of lipid nanoparticles(LNPs)and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu   +6 more
wiley   +1 more source

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