Results 31 to 40 of about 113,622 (237)

Bridging the Gap in Neuroblastoma Care: Consensus‐Based Statements With Recommendations for Improved Patient and Caregiver Experiences

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Neuroblastoma's complex, heterogeneous biology poses significant diagnostic and therapeutic challenges, often requiring caregivers to absorb complex information and participate in time‐sensitive decisions. However, caregivers often feel unprepared to evaluate options.
Vickie Buenger, Joseph Bauer, Jennifer Diamond, Maggie Fader, Shae Hansen, Jacqueline M. Kraveka, Johanna Schoen, Amber Shearer, Dena Sherwood   +8 more
wiley   +1 more source

Production of therapeutic levels of human FIX-R338L by engineered B cells using GMP-compatible medium

Molecular Therapy: Methods & Clinical Development, 2023
B cells can differentiate into plasmablast and plasma cells, capable of producing antibodies for decades. Gene editing using zinc-finger nucleases (ZFN) enables the engineering of B cells capable of secreting sustained and high levels of therapeutic ...
Marion David, Davide Monteferrario, Gaëlle Saviane, Caroline Jeanneau, Irène Marchetti, Coralie F. Dupont, Céline Dumont, Jason D. Fontenot, Maurus de la Rosa, David Fenard   +9 more
doaj   +1 more source

Phase Angle as an Early Functional Biomarker of Cancer‐Related Fatigue in Pediatric Oncology: A Prospective Longitudinal Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Pediatric cancer remains a leading cause of morbidity and mortality worldwide, particularly in low‐and middle‐income countries. Cancer treatment may impair nutritional status, alter body composition, and exacerbate cancer‐related fatigue (CRF).
Luís Carlos Lopes‐Junior, Luiz Claudio Barreto Silva Neto, Fabiano Kenji Haraguchi, Letícia Batista de Azevedo, Marcelo Brenner Nascimento Carlini, Raphael Manhães Pessanha, Mariana Rabello Laignier, Wesley Rocha Grippa, Tania Mara Lopes Bitti Bortolini, Gláucia Zouain‐Figueiredo, Regina Aparecida Garcia de Lima, Liliana Vasquez   +11 more
wiley   +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

Purificación del factor IX recombinante expresado en leche de cerdas transgénicas Purification of recombinant factor IX expressed in milk of transgenic sows

Revista Cubana de Hematología, Inmunología y Hemoterapia, 2006
Se purificó a partir de la leche de cerdas transgénicas, factor IX recombinante, y se obtuvieron rendimientos entre 1 a 2 g de esta proteína por litro, lo que resulta una nueva vía para la obtención de este producto con una alta eficiencia, ya que su ...
Armando Cádiz Lahens, Aniel Moya Torres, William Velander, Manuel Limonta Vidal, Lázaro Joo Sánchez, Alexandro Rodríguez Rojas, Laritza Solarana Tito, Alina Díaz Concepción, Patricia Caunedo Almagro, Dunia Castillo González, Delfina Almagro Vázquez   +10 more
doaj  

CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs

Haematologica, 2020
Hemophilia B is an X-linked recessive bleeding disorder caused by abnormalities in the coagulation factor IX gene. Without prophylactic treatment, patients experience frequent spontaneous bleeding episodes.
Jiahuan Chen, Beiying An, Biao Yu, Xiaohuan Peng, Hongming Yuan, Qiangbing Yang, Xue Chen, Tingting Yu, Lingyu Wang, Xinwei Zhang, He Wang, Xiaodong Zou, Daxin Pang, Hongsheng Ouyang, Xiaochun Tang   +14 more
doaj   +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

Encapsulation of factor IX–engineered mesenchymal stem cells in fibrinogen–alginate microcapsules enhances their viability and transgene secretion

Journal of Tissue Engineering, 2012
Cell microencapsulation holds significant promise as a strategy for cellular therapies; however, inadequate survival and functionality of the enclosed cells limit its application in hemophilia treatment.
Bahareh Sayyar, Megan Dodd, Jianping Wen, Shirley Ma, Leah Marquez-Curtis, Anna Janowska-Wieczorek, Gonzalo Hortelano   +6 more
doaj   +1 more source

F9 Malmö, factor IX and deep vein thrombosis

Haematologica, 2009
Background We recently reported the association between the Malmö sequence variant in F9 (rs6048) and deep vein thrombosis.Design and Methods We aimed to study whether the association between F9 Malmö and deep vein thrombosis is explained by linkage ...
Irene D. Bezemer, Andre R. Arellano, Carmen H. Tong, Charles M. Rowland, Helen A. Ireland, Kenneth A. Bauer, Joseph Catanese, Pieter H. Reitsma, Carine J.M. Doggen, James J. Devlin, Frits R. Rosendaal, Lance A. Bare   +11 more
doaj   +1 more source

Occurrence of Extramedullary Relapses in Pediatric Acute Lymphoblastic Leukemia After Treatment With Blinatumomab

Pediatric Blood &Cancer, EarlyView.
ABSTRACT We report a retrospective single‐center analysis of pediatric patients with relapsed or refractory B‐cell precursor acute lymphoblastic leukemia focusing on relapses outside of the typical locations, bone marrow, central nervous system, or testes.
Johanna Kunz, Martin Ebinger, Michaela Döring, Markus Mezger, Ursula Holzer, Rupert Handgretinger, Peter Lang, Manon Queudeville   +7 more
wiley   +1 more source