Results 51 to 60 of about 10,746,438 (360)

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Science Advances, 2020
Editing the fetal γ-globin promoters in hematopoietic stem cells from sickle cell disease patients induces therapeutic γ-globin levels. Sickle cell disease (SCD) is caused by a single amino acid change in the adult hemoglobin (Hb) β chain that causes Hb ...
Leslie Weber, G. Frati, T. Felix, G. Hardouin, Antonio Casini, Clara Wollenschlaeger, Vasco Meneghini, C. Masson, A. de Cian, Anne Chalumeau, F. Mavilio, Mario Amendola, I. André-Schmutz, A. Cereseto, W. El Nemer, J. Concordet, C. Giovannangeli, M. Cavazzana, A. Miccio   +18 more
semanticscholar   +1 more source

HSP70 governs permeability and mechanotransduction in primary human endothelial cells

FEBS Open Bio, EarlyView.
HSP70 chemical inhibition reduces endothelial cell proliferation and increases permeability, the latter supported by normal interendothelial junctional protein distribution. HSP70 also plays a role in shear stress response, a hemodynamic force naturally present in blood vessels and correlated with vessel protection.
Andrea Pinto‐Martinez, Everton G. Melo, Isadora C. B. Pavan, Percíllia V. S. Oliveira, Luiza B. C. T. Coimbra, Thaís L. S. Araujo   +5 more
wiley   +1 more source

Cognitive Behavioral Therapy for Youth with Childhood‐Onset Lupus: A Randomized Clinical Trial

Arthritis Care &Research, Accepted Article.
Objective Our objective was to determine the feasibility and acceptability of the Treatment and Education Approach for Childhood‐onset Lupus (TEACH), a six‐session cognitive behavioral intervention addressing depressive, fatigue, and pain symptoms, delivered remotely to individual youth with lupus by a trained interventionist.
Natoshia R. Cunningham, Thea Senger‐Carpenter, Jocelyn Zuckerman, Michelle Adler, Mallet R. Reid, Ashley Danguecan, Luana Flores Pereira, Sarah I. Mossad, Samantha L. Ely, Khalid Abulaban, Elizabeth A. Kessler, Natalie Rosenwasser, Tamar B. Rubinstein, Ekemini A. Ogbu, Emily A. Smitherman, Alaina Miller, Taylor Abounader, Elizabeth Ross, Livie Timmerman, Dhriti Sharma, Jennifer N. Stinson, Stacy Allen, Kabita Nanda, Tala El Tal, Deborah M. Levy, Linda T. Hiraki, Hermine I. Brunner, Mathew Reeves, Steven J. Pierce, Andrea Knight   +29 more
wiley   +1 more source

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

Treating sickle cell anemia

Science, 2020
New drugs, stem cell transplants, and gene therapy show promise in treating sickle cell anemia Sickle cell anemia is an inherited disorder caused by a point mutation (affecting a single nucleotide) in the gene that encodes the β-globin chain of ...
J. Tisdale, S. Thein, W. Eaton
semanticscholar   +1 more source

Discovery of a Novel DNMT1 Inhibitor with Improved Efficacy in Treating β‐Thalassemia

Advanced Science, EarlyView.
Context of Research: β‐thalassemia affects millions worldwide. DNMT inhibitors are effective HbF‐inducers that benefit patients with β‐thalassemia. Existing DNMT inhibitors are not approved for β‐thalassemia treatment due to dose‐limiting toxicity.What We Find: DMT207 traps DNMT1 into helix‐kinked inactive conformation and enhances its interaction with
Yijie Shen, Jiale Wei, Shibing Tang, Dongliang Wu, Liangyi Zong, Shuyuan Ma, Qing Xiong, Ruijie Gong, Siyuan Xu, Chuxuan Peng, Qin Feng, Songchen Liu, Qitong Liu, Yuhua Ye, Quan Zhao, Cheng Luo, Peng Huang, Zhihai Li, Xiangqian Kong, Xianjiang Lan   +19 more
wiley   +1 more source

Acute Exercise Challenge and Airway Dynamics in Youth With Sickle Cell Anemia: A Multicenter Study

American Journal of Hematology, EarlyView.
Changes in airway dynamics in children with sickle cell anemia after maximal cardiopulmonary exercise testing and a controlled intensity interval excercise challenge. ABSTRACT Sickle cell anemia (SCA) leads to reduced physical functioning and cardiopulmonary fitness. Prior studies suggest that airway hyperresponsiveness to bronchoprovocation testing is
Robyn T. Cohen, Jane S. Hankins, Kirsten K. Ness, Lewis L. Hsu, Tracy Baynard, Amy Tang, Shlomit Radom‐Aizik, Kevin Guerrero, Mark Rodeghier, Robert I. Liem   +9 more
wiley   +1 more source

Inheritance of Hereditary Persistence of Fetal Haemoglobin (HPFH) in a Family of Western Odisha, India [PDF]

Journal of Clinical and Diagnostic Research, 2015
Hereditary persistence of foetal haemoglobin (HPFH) is a rare inherited haemoglobin disorders in India. We encountered five cases of HPFH-3 in heterozygous condition in a single family of western Odisha, India. All the cases had raised % HbF (26.1±3.23%)
Siris Patel, Snehadhini Dehury, Prasanta Purohit, Satyabrata Meher, Kishalaya Das   +4 more
doaj   +1 more source

Extra-Medullary Hematopoiesis in Sickle Cell Disease Presenting as a Right Adrenal Mass [PDF]

, 2022
Folasade Ajayi, Meenakshi S Nali, Ruhma Ali, Aditya Patel, Hamid Shaaban   +4 more
openalex   +1 more source

Estimated Life Expectancy and Income of Patients With Sickle Cell Disease Compared With Those Without Sickle Cell Disease

JAMA Network Open, 2019
Key Points Question What is the association between sickle cell disease and life expectancy and lifetime income? Findings This cohort simulation modeling study showed that projected life expectancy (54 vs 76 years) and quality-adjusted life expectancy ...
D. Lubeck, I. Agodoa, N. Bhakta, M. Danese, Kartik S. Pappu, Robin S. Howard, M. Gleeson, M. Halperin, S. Lanzkron   +8 more
semanticscholar   +1 more source