Results 141 to 150 of about 920,848 (226)

Genome Editing for Glycogen Storage Diseases

Journal of Inherited Metabolic Disease, Volume 49, Issue 5, September 2026.
ABSTRACT Gene therapy has been developed for several glycogen storage diseases and has advanced into clinical trials. However, the limitations of these gene therapies with regard to stability following treatment early in life have led to the development of genome editing.
Troy von Beck, Raymond Wang, Dwight Koeberl   +2 more
wiley   +1 more source

Depression in mothers of children with thalassemia or blood malignancies: a study from Iran [PDF]

Background Several studies have found that parents of children with chronic diseases or disabilities have higher depression scores than control parents. Mothers usually take on the considerable part of the extra care and support that these children need
AliReza Farrokhi, کاربخش, مژگان, میثمی, علی پاشا, Mojgan Karbakhsh, نبئی , بهروز, Behrooz Nabaei, Alipasha Meysamie, فرخی, علیرضا, شرقی, افشان, Afshan Sharghi   +9 more
core   +1 more source

Cerebral Blood Transit in Sickle Cell Anemia

Journal of Magnetic Resonance Imaging, Volume 64, Issue 3, Page 738-750, September 2026.
ABSTRACT Background Sickle cell anemia (SCA) patients upregulate cerebral blood flow to compensate for decreased arterial oxygen content. Such hyperemic conditions can manifest as venous hyperintense signal on arterial spin labeling (ASL) MRI, which may reflect faster capillary blood transit, altered oxygen extraction fraction (OEF), and infarct risk ...
Wesley T. Richerson, Megan A. Aumann, Alexander K. Song, Samantha Davis, Lauren Milner, Maria Garza, Dann Martin, L. Taylor Davis, Sumit Pruthi, Lori C. Jordan, Manus J. Donahue   +10 more
wiley   +1 more source

Sickle Cell Disease and Kidney Injury: Circulating Uromodulin Allows Early Tissue Specific Diagnosis and Monitoring of Treatment

American Journal of Hematology, Volume 101, Issue 9, Page 2430-2434, September 2026.
Ferras Alashkar, Robert Hable, Denise Zwanziger, Friederike Poppenborg, Raina Yamamoto, Hans Christian Reinhardt, Alexander Röth, Victor Herbst, Yara Bestmann, Marco Kai, Jürgen E. Scherberich   +10 more
wiley   +1 more source

Smart Design: Integrating Artificial Intelligence and Gene Editing for Advanced mRNA Therapeutics

MedComm – Biomaterials and Applications, Volume 5, Issue 3, September 2026.
The challenges of mRNA therapy and the application of artificial intelligence and gene editing in the field of mRNA drugs. ABSTRACT Artificial intelligence (AI) and gene editing are increasingly being applied to the design and evaluation of mRNA therapeutics.
Haixing Shi, Shengbin Liu, Danni Dai, Shanhui Jiang, Tingchen Tian, Zhenyi Niu, Zhongzheng Xiang, Chaoyu Zou, Fang Liu, Xue Tang, Xin Jiang, Xiangrong Song   +11 more
wiley   +1 more source

Blood rheology in children with the S/β+-thalassemia syndrome

, 2018
International audienceThe aim of the present study was to compare blood rheological parameters between children with homozygous sickle cell disease (SS), sickle cell SC disease or S/β+-thalassemia syndrome, and healthy children (AA) and to test the ...
Gauthier, Alexandra, Romanet-Faes, Camille, Joly, Philippe, Pialoux, Vincent, Yves Bertrand, Alexandra Gauthier, Camille Romanet-Faes, Philippe Joly, Daniela Cuzzubbo, Garnier, Nathalie, Renoux, Céline, Cannas, Giovanna, Céline Renoux, Vincent Pialoux, Nathalie Garnier, Bertrand, Yves, Connes, Philippe, Cuzzubbo, Daniela, Giovanna Cannas, Philippe Connes   +19 more
core   +1 more source

DAMPs, PAMPs, and Alarmins: From Mechanism to Therapy

MedComm, Volume 7, Issue 9, September 2026.
This review synthesizes the expanding biology of PAMPs and DAMPs, from their molecular sources and sensing mechanisms to regulatory networks that govern inflammation and homeostasis. Spanning from classical immune activation to trained immunity and interorgan communication, we highlight the interplay between PAMPs and DAMPs and their context‐dependent ...
Xuanxuan Yu, Yuqin Jin, Baochao Li, Jie Deng, Yiwen Zhou, Jinglun Zhang, Huang Li   +6 more
wiley   +1 more source

Mirror Syndrome (Ballantyne Syndrome): Prenatal Diagnosis, Pathophysiology, and the Role of Fetal Therapy—A Narrative Review

Prenatal Diagnosis, Volume 46, Issue 10, Page 1617-1625, September 2026.
ABSTRACT Mirror syndrome is a rare maternal–fetal condition associated with fetal hydrops and a high risk of adverse maternal and fetal perinatal outcomes. Its diagnosis is challenging due to the lack of standardized diagnostic criteria and its clinical and biochemical overlap with preeclampsia.
Riccardo Tudisco, Pauline Latapie, Anita Romiti, Alexandra Benachi, Maelig Abgral, Alexandre J. Vivanti   +5 more
wiley   +1 more source

Molecular and hematological characterization of thalassemia and hemoglobinopathies among pediatric patients in northern Lao People’s Democratic Republic

Scientific Reports
Thalassemia and hemoglobinopathies are highly prevalent in the Lao People’s Democratic Republic (Lao PDR). Luang Prabang Province represents an ethnically diverse area with a high burden of pediatric anemia, but the molecular and hematological ...
Volapheth Kanyasone, Pichpimon Pakpuak, Supitsaraporn Satewin, Anupong Pansuwan, Kritsada Singha, Attawut Chaibunruang, Hataichanok Srivorakun, Supan Fucharoen, Annkham Thammaseng, Sengmany Sengphachan, Hame Thiphalack, Anousine Phonedala, Supawadee Yamsri   +12 more
doaj   +1 more source

Hydroxyurea (hydroxycarbamide) use in adults with haemoglobin SC disease: A real‐world study in Quebec

British Journal of Haematology, Volume 209, Issue 3, Page 1291-1300, September 2026.
Summary Haemoglobin SC (HbSC) disease is the second most prevalent form of sickle cell disease, but evidence for hydroxyurea (hydroxycarbamide; HU) to prevent pain episodes was limited until the prospective identification of variables as outcomes for treatment (PIVOT) trial.
Alice Girard, Laurianne Charette, Mélissa Haouchine, Laurence Bozec, Mohamed Zakaria Bachir Bouyadjera, Mariane Poitras, Vincent‐Thierry Taillefer, Chloé Trudeau, Yves Pastore, Nathalie Letarte, Stéphanie Forté   +10 more
wiley   +1 more source