Results 41 to 50 of about 1,304,324 (179)

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, EarlyView.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source

Identification of epigenetic signature associated with alpha thalassemia/mental retardation X-linked syndrome

Epigenetics & Chromatin, 2017
Alpha thalassemia/mental retardation X-linked syndrome (ATR-X) is caused by a mutation at the chromatin regulator gene ATRX. The mechanisms involved in the ATR-X pathology are not completely understood, but may involve epigenetic modifications.
L. Schenkel, K. Kernohan, A. McBride, Ditta Reina, A. Hodge, P. Ainsworth, D. Rodenhiser, G. Paré, N. Bérubé, C. Skinner, K. Boycott, C. Schwartz, B. Sadikovic   +12 more
semanticscholar   +1 more source

Loss of SOCS1 in Donor T Cells Exacerbates Intestinal GVHD by Driving a Chemokine‐Dependent Pro‐Inflammatory Immune Microenvironment

Advanced Science, EarlyView.
T cell‐specific Socs1 knockout leads to inflammatory differentiation of CD8+ T cells, prompting the STAT1/2 complex to drive the activation of Ccl5, Ccr5, and Cxcr3, and promoting the skewing of monocytes toward a pro‐inflammatory M1 macrophage lineage.
Zhigui Wu, Bixia Wang, Xinya Jiang, Fangqing Zhang, Qianqian Huang, Xinyi Wu, Shuang Fan, Qi Zhang, Jingrui Zhou, Jianing Tang, Xiao‐Dong Mo, Yu Wang, Ying‐Jun Chang, Huidong Guo, Xiao‐Jun Huang   +14 more
wiley   +1 more source

Alpha globin gene alterations modifying the phenotype of homozygous beta thalassaemia

eJHaem
The phenotype of β‐thalassemia varies widely. The primary determinant is the type of beta‐globin gene mutation; however, there are secondary and tertiary modifiers also as associated alpha mutations, polymorphisms, as well as coinheritance of mutations ...
Jyoti Shaw, Abhilipsa Patra, Anjumana Khatun, Rudra Ray, Amit Ghosh, Sonali Mahapatra, Ashutosh Panigrahi, Maitreyee Bhattacharyya   +7 more
doaj   +1 more source

Alpha-Thalassemia in North Morocco: Prevalence and Molecular Spectrum

BioMed Research International, 2019
Unlike the other hemoglobinopathies, few researches have been published concerning α-thalassemia in Morocco. The epidemiological features and the mutation spectrum of this disease are still unknown. This regional newborn screening is the first to study α-
Achraf Laghmich, Fatima Zahra Alaoui Ismaili, A. Barakat, Naima Ghailani Nourouti, M. Khattab, Mohcine Bennani Mechita   +5 more
semanticscholar   +1 more source

Altered Brain Structure in an ATRX‐Deficient Mouse Model of Autism Spectrum Disorder

Autism Research, EarlyView.
ABSTRACT Mutations in the ATRX gene are a primary cause of alpha‐thalassemia intellectual disability X‐linked (ATRX) syndrome, which is characterized by intellectual disability, autism, and a range of brain structural abnormalities, including microcephaly.
Katherine Quesnel, Jacob Ellegood, Jason P. Lerch, Nathalie G. Bérubé   +3 more
wiley   +1 more source

Survival and causes of death in patients with alpha and beta-thalassemia in Northern Thailand

Annals medicus
Background Thalassemia is the most prevalent hereditary anaemia worldwide. Severe forms of thalassemia can lead to reduced life expectancy due to disease-related complications. Objectives To investigate the survival of thalassemia patients across varying
A. Tantiworawit, Thansita Kamolsripat, P. Piriyakhuntorn, T. Rattanathammethee, S. Hantrakool, C. Chai-Adisaksopha, E. Rattarittamrong, L. Norasetthada, K. Fanhchaksai, P. Charoenkwan   +9 more
semanticscholar   +1 more source

Alpha- Thalassemia: An Overview

Scholars Journal of Applied Medical Sciences, 2023
Alpha-Thalassemia is the commonest hereditary monogenic disease worldwide. α-thalassemia is caused by α-globin gene losses and categorized into α-thalassemia 1 and α-thalassemia 2 depending on how many α-globin genes are functioning. Α thalassemia 1 is defined by inactivation of both α-globin genes on a chromosome, while in α-thalassemia 2, one gene is
openaire   +1 more source

Nano‐networks via reaction‐induced self‐assembly coordinate spatiotemporal multi‐drug delivery for acute kidney injury therapy

BMEMat, EarlyView.
Multifunctional nano‐networks (NNWs) are prepared via reaction‐induced self‐assembly (RISA) of spermidine (SPD), epigallocatechin gallate (EGCG), 2‐formylphenylboronic acid (2‐FPBA), and deferoxamine (DFO) through dynamic iminoboronate bonds. These NNWs leverage the oxidative AKI microenvironment to trigger disintegration, enabling site‐specific ...
Juan Jin, Xiao Zhang, Chenxi Wang, Taian Jin, Xinyang Fang, Yixuan Gao, Mengchun Chen, Weiyang Meng, Chang Gao, Wenlu Li, Yuanfeng Li, Yijie Chen, Calvin A. Omolo, Thirumala Govender, Linqi Shi, Yunguang Wang, Yong Liu, Qiang He   +17 more
wiley   +1 more source

Prevalence of Co-Inheritance of Alpha-Thalassemia with Beta-Thalassemia and Beta-Hemoglobinopathy in Ahvaz City

Journal of Ardabil University of Medical Sciences, 2013
Background: Co-inheritance of hemoglobin gene defects is a rare important status that can lead to double heterozygote or homozygote with significant clinical manifestations. Such conditions can be observed in co-inheritance of alpha-thalassemia with beta-
Najmaddin Saki, Akbar Dorgalaleh, Zahra Kashani Khatib, Shaban Alizadeh, Fakher Rahim, Hamid Galehdari, Bijan Kaikhaei, Mohammad Pedram, Ali Dehghani Fard   +8 more
doaj