LDB1 represses fetal hemoglobin expression by enhancing BCL11A transcription. [PDF]
Park SW +9 more
europepmc +1 more source
Engineering Biology Beyond Single Genes: Advances and Challenges in Multiplex Genome Editing
Multiplex genome editing is transforming genome engineering from single‐gene perturbation to network‐level control, yet its broader application remains limited by challenges in gRNA array engineering, delivery technologies, and safety management. Emerging AI‐driven approaches are accelerating guide RNA design and CRISPR effector optimization for ...
Linli Wang, Yongbin Liu, Hongbing Han
wiley +1 more source
Targeted protein degradation for fetal hemoglobin induction: a new paradigm in β-hemoglobinopathy therapy. [PDF]
Arji S +12 more
europepmc +1 more source
Accurate prediction of fetal hemoglobin by Doppler ultrasonography
OBJECTIVE: To assess the feasibility of using the middle cerebral artery peak systolic velocity (MCA-PSV) to predict the actual value of fetal hemoglobin in fetuses undergoing a first cordocentesis for detection of anemia caused by maternal red cell ...
Oz, U. +5 more
core
Human Monocytic Models Reveal Genotype‐Dependent Inflammatory Programs in VEXAS Syndrome
Objectives VEXAS syndrome is a severe X‐linked autoinflammatory disorder caused by somatic mutations in ubiquitin‐like modifier activating enzyme 1 (UBA1), with clinical outcomes that vary by UBA1 genotype. We aimed to elucidate genotype‐specific inflammatory programs and identify potential therapeutic targets.
Kana Higashitani +10 more
wiley +1 more source
PGC-1α agonism via oral administration of ZLN005 induces fetal hemoglobin and is antisickling in sickle mice. [PDF]
Benmhammed H +16 more
europepmc +1 more source
Precision medicine in paediatrics: Progress and priorities
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
STUDIES ON THE FETAL HEMOGLOBIN OF RABBITS
TYUMA, Itiro +2 more
openaire +3 more sources
Stroke From Paradoxical Embolism in a Young Patient With Sickle-Cell Disease and High Fetal Hemoglobin: A Diagnostic Challenge. [PDF]
Luo YH +5 more
europepmc +1 more source
Fetal hemoglobin seviyesinin orak hücre anemisi hastalarının kemik biyokimyası üzerine etkisi
Amaç: Bu çalışmanın amacı fetal hemoglobin seviyelerinin, orak hücre anemisi hastalarının kemik parametreleri üzerine olası etkisinin değerlendirilmesidir.Metod: 56'sı SS, 47'si kontrol grubu olmak üzere 103 kan örneği, fetal hemoglobin düzeylerine göre ...
Zeliha Nazan Alparslan +4 more
core

