Results 61 to 70 of about 1,336,426 (267)

Establishment of an α-thalassemia mouse model through fetal liver cell transplantation and analysis of hematological parameters

Scientific Reports
Clinically, α-thalassemia is stratified into mild, intermediate, and severe forms, differentiated by the degree of anemia severity. Specifically, severe α-thalassemia manifests in homozygous individuals, characterized by a profound α globin deficit.
Xin Xu, Wencheng Fu, Wenrui Ye
doaj   +1 more source

A Comprehensive Study of the Effects by Sequence Truncation within Inverted Terminal Repeats (ITRs) on the Productivity, Genome Packaging, and Potency of AAV Vectors

Microorganisms
One of the primary challenges in working with adeno-associated virus (AAV) lies in the inherent instability of its inverted terminal repeats (ITRs), which play vital roles in AAV replication, encapsidation, and genome integration.
Yinxing Chen, Shiliang Hu, William Lee, Noel Walsh, Kayla Iozza, Neil Huang, Gregory Preston, Lauren M. Drouin, Nannan Jia, June Deng, Matthias Hebben, Jing Liao   +11 more
doaj   +1 more source

Induction of Diabetes Abolishes the Antithrombotic Effect of Clopidogrel in Apolipoprotein E–Deficient Mice

TH Open, 2017
Patients with acute coronary syndrome with diabetes mellitus (DM) exhibit an impaired platelet inhibitory response to clopidogrel which is only partially understood. DM was induced by the administration of streptozotocin (STZ) to 9-week-old mice.
A. Sugidachi, K. Ohno, J. A. Jakubowski, Y. Ito, A. Tomizawa, M. Mizuno   +5 more
doaj   +1 more source

Ovarian Sex Cord Stromal Tumors in Children and Adolescents—The European Standard Clinical Practice Recommendations

Pediatric Blood &Cancer, EarlyView.
ABSTRACT As part of the European Cooperative Study Group for Paediatric Rare Tumours initiative, we developed standard clinical practice guidelines for ovarian sex cord stromal tumors, based on comprehensive national and international cohort analyses, literature review, and a final expert consensus conference.
Dominik T. Schneider, Kris‐Ann Schultz, Nora Schnitzler, Michael Abele, Tal Ben‐Ami, Ewa Bien, Gianni Bisogno, Ines B. Brecht, Maja Cesen, Andrea Ferrari, Michaela Kuhlen, Malgorzata A. Krawczyk, Ricardo Lopez‐Almaraz, Yves Reguerre, Calogero Virgone, Daniel Orbach   +15 more
wiley   +1 more source

Natural Killer Cells in Paediatric Soft Tissue Sarcomas: A Systematic Review

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Paediatric soft tissue sarcomas (pSTS) are a rare and heterogeneous group of malignant tumours arising in tissues of mesenchymal origin. The role of natural killer (NK) cells in pSTS remains poorly understood, with evidence fragmented across small preclinical studies and early‐phase clinical trials.
Raya Dean, Lara V. Graham, Christina Putnam, Rosa Gomes Alves Martins, Daniel T. Harding, Tim J. Underwood, Zoë S. Walters, Matthew D. Blunt   +7 more
wiley   +1 more source

Can patient self-evaluation of functional status be used for evaluation of impairment of motor function in Guillain-Barré syndrome? Mapping clinician- and patient-reported outcomes in a phase 3 study of eculizumab in Japan

Frontiers in Neurology
Background and purposeGuillain-Barré syndrome (GBS) is an autoimmune neurological disorder characterized by muscle weakness. In clinical trials, treatment benefit and disease severity are typically measured using clinician-reported outcome measures like ...
Antoine Regnault, Angély Loubert, Stéphane Quéré, Qun Lin, Glen Frick, Hirokazu Ishida, Yuko Abeta, Helene Chevrou-Severac   +7 more
doaj   +1 more source

A Population‐Based Study on Childhood Aplastic Anemia—Incidence, Outcomes, and Health‐Related Quality of Life

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Childhood aplastic anemia (AA) is a rare disease, and both the disease itself and its treatment cause significant morbidity. We aimed to determine the contemporary incidence of childhood AA in Finland, to compare the clinical characteristics of AA against inherited bone marrow failure syndromes (IBMFS) and refractory cytopenia of ...
Lauri‐Matti Kulmala, Riitta Niinimäki, Tytti Pokka, Anu Huurre, Liisa Järvelä, Sauli Palmu, Tuuli Pöyhönen, Pauliina Utriainen, Henri Aarnivala   +8 more
wiley   +1 more source

The Role of Chemotherapy in Pediatric Myoepithelial Carcinoma: A Systematic Review of the Literature

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Myoepithelial carcinoma (MEC) in pediatric patients is a rare and aggressive malignancy characterized by heterogeneous morphology and variable molecular features. The optimal role of chemotherapy remains unclear. We conducted a systematic review according to PRISMA 2020 guidelines to evaluate chemotherapy in pediatric and young‐adult patients ...
Marco Salvi, Maria Carmen Affinita, Giulia Fichera, Luisa Santoro, Angela Scagnellato, Calogero Virgone, Elena Poli, Gianni Bisogno   +7 more
wiley   +1 more source

Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency

Communications Medicine
Background Multiple Sulfatase Deficiency (MSD) is a rare inherited lysosomal storage disorder characterized by loss of function mutations in the SUMF1 gene that manifests as a severe pediatric neurological disease.
Maximiliano Presa, Rachel M. Bailey, Somdatta Ray, Lauren Bailey, Saurabh Tata, Tara Murphy, Pierre-Alexandre Piec, Harold Combs, Steven J. Gray, Cathleen Lutz   +9 more
doaj   +1 more source

Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice

Molecular Therapy: Methods & Clinical Development
Pompe disease (PD) is a multisystemic progressive disease caused by acid-alpha glucosidase (GAA) deficiency. Patients display a spectrum of phenotypes ranging from the severe, rapidly progressive infantile-onset PD (IOPD) form to the slower progressing ...
Kelly George, Raquel Riley, Shan Zhou, Elizabeth Allen, Laurie Smith, Elena Kistanova, Sofia Kinton, Lilu Guo, Dinesh Bangari, Ayman Ismail, Jayaprakash Thummapudi, Fawzi Boumezbeur, Erwan Selingue, Dorothée Tamarelle, Cecile Capdevila, Tuan-Minh Do, Dominique Lesuisse, Nina Leksa, Pablo Sardi, Arjan van der Flier   +19 more
doaj   +1 more source