Results 131 to 140 of about 82,601 (269)

Epigenetic modulations on the fetal hemoglobin induction [PDF]

open access: yesInternational Journal of Hematology-Oncology and Stem Cell Research, 2012
In recent years of experience fetal hemoglobin (HbF) induction considers as novel therapeutic approach for β-thalassemia and sickle cell in therapeutic approaches.
Najmaldin Saki   +4 more
doaj  

Longitudinal Analysis of Sleep‐Disordered Breathing and Cognitive Outcomes in Children Living With Sickle Cell Anaemia

open access: yesClinical Otolaryngology, Volume 51, Issue 4, Page 520-528, July 2026.
ABSTRACT Objectives Sleep‐disordered breathing (SDB) and cognitive challenges are commonly observed in children living with sickle cell anaemia (SCA). This study investigated the longitudinal change in polysomnographic outcomes and the association with cognitive functions in children living with SCA.
Shifa Hamdule   +5 more
wiley   +1 more source

Patient‐Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non‐Transfusion‐Dependent β‐Thalassemia Treated in the BEYOND Trial

open access: yesEuropean Journal of Haematology, Volume 117, Issue 1, Page 161-173, July 2026.
ABSTRACT In the phase 2, double‐blind, randomized controlled BEYOND trial (NCT03342404), luspatercept increased hemoglobin levels in patients with non‐transfusion‐dependent β‐thalassemia (NTDT). This study assessed long‐term effects of luspatercept on patient‐reported outcomes (PROs), using data from BEYOND and patients who continued luspatercept ...
Khaled M. Musallam   +12 more
wiley   +1 more source

Comparative analysis of globin loci in pufferfish and man suggests a common origin of vertebrate globin loci and reveals a novel mammalian globin locus.

open access: yes, 2003
To further our understanding of the regulation of vertebrate globin loci, we have isolated cosmids containing - and -globin genes from the pufferfish Fugu rubripes.
Burgtorf, Carola   +11 more
core  

Genetic attenuation of ALDH1A1 increases metastatic potential and aggressiveness in colorectal cancer

open access: yesMolecular Oncology, Volume 20, Issue 6, Page 1556-1590, June 2026.
Aldehyde dehydrogenase 1A1 (ALDH1A1) is a cancer stem cell marker in several malignancies. We established a novel epithelial cell line from rectal adenocarcinoma with unique overexpression of this enzyme. Genetic attenuation of ALDH1A1 led to increased invasive capacity and metastatic potential, the inhibition of proliferation activity, and ultimately ...
Martina Poturnajova   +25 more
wiley   +1 more source

Epigenetic interplay at the β-globin locus

open access: yes, 2017
During development, the α- and β-globin genes exhibit a highly conserved pattern of expression, giving rise to several developmental stage-specific hemoglobin variants.
McColl, B   +3 more
core   +1 more source

A Cryoprotectant‐Compatible Nanoporous Platform for Stable and Scalable Delivery of Biopharmaceuticals

open access: yesAdvanced Materials, Volume 38, Issue 33, 12 June 2026.
A cryoprotectant‐compatible nanoporous platform enables ambient‐stable and scalable delivery of gene editing therapeutics. By combining hierarchical pore architecture with optimized lyophilization chemistry, the system preserves Cas9‐RNP activity post‐freeze‐drying.
Sian Lee   +8 more
wiley   +1 more source

Modification of globin gene expression by RNA targeting strategies.

open access: yes, 2018
OBJECTIVE: Sickle cell anemia is a genetic blood disease resulting from production of mutant beta-globin (beta(S)) and has severe clinical consequences. It is known that a higher cellular gamma-globin level, e.g., higher ratio of cellular gamma-globin to
Tong-Jian Shen (5062436)   +5 more
core   +1 more source

A Central Somatic Transmission Mediates Proprioceptive Facilitation of Muscle Pain

open access: yesAdvanced Science, Volume 13, Issue 34, 19 June 2026.
Zhang et al. uncover a novel central mechanism for persistent muscle pain, in which TRPA1 sensitization in MeV proprioceptive neurons enhances somatic secretion. This, in turn, disinhibits descending pain control from neighboring noradrenergic locus coeruleus neurons via local GABAergic circuits, thereby promoting inflammatory muscle pain.
Xiaoyu Zhang   +15 more
wiley   +1 more source

shRNA screen for novel epigenetic regulators of γ-globin silencing [PDF]

open access: yes, 2016
© 2016 Dr. Betty Rui-Yun Kaoβ-thalassemia is a common blood disorder caused by mutations within the β-globin gene that result in insufficient hemoglobin production, reduced red blood cell half-life and life-threatening anemia. High expression of γ-globin
Kao, Betty Rui-Yun
core  

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