Results 11 to 20 of about 193,098 (314)

Primary extranodal diffuse large B‐cell lymphoma: Molecular features, treatment, and prognosis

Aging and Cancer, Volume 3, Issue 3-4, Page 133-146, September-December 2022., 2022
One‐third of DLBCL arises from extranodal organs and is challenging to manage. Molecular features are critical to elucidate the differences in clinical features, predict the disease prognosis, and improve effective targeted therapeutic strategies. Abstract Diffuse large B‐cell lymphoma (DLBCL) is the most common subtype of non‐Hodgkin's lymphoma and ...
Si‐Yuan Chen, Meng‐Meng Ji, Peng‐Peng Xu, Wei‐Li Zhao   +3 more
wiley   +1 more source

Phenotypic continuum of NFU1‐related disorders

Annals of Clinical and Translational Neurology, Volume 9, Issue 12, Page 2025-2035, December 2022., 2022
Abstract Bi‐allelic variants in Iron–Sulfur Cluster Scaffold (NFU1) have previously been associated with multiple mitochondrial dysfunctions syndrome 1 (MMDS1) characterized by early‐onset rapidly fatal leukoencephalopathy. We report 19 affected individuals from 10 independent families with ultra‐rare bi‐allelic NFU1 missense variants associated with a
Rauan Kaiyrzhanov, Maha S. Zaki, Tracy Lau, Sambuddha Sen, Reza Azizimalamiri, Mina Zamani, Gözde Yeşil Sayin, Taru Hilander, Stephanie Efthymiou, Viorica Chelban, Ruth Brown, Kyle Thompson, Maria Irene Scarano, Jaya Ganesh, Kairgali Koneev, Ismail Musab Gülaçar, Richard Person, Dinara Sadykova, Yerdan Maidyrov, Tahereh Seifi, Aizhan Zadagali, Geneviève Bernard, Katrina Allis, Houda Zghal Elloumi, Amanda Lindy, Ehsan Taghiabadi, Sumit Verma, Rachel Logan, Brian Kirmse, Renkui Bai, Shaimaa M. Khalaf, Mohamed S. Abdel‐Hamid, Alireza Sedaghat, Gholamreza Shariati, Mahmoud Issa, Jawaher Zeighami, Hasnaa M. Elbendary, Garry Brown, Robert W. Taylor, Hamid Galehdari, Joseph J. Gleeson, Christopher J. Carroll, James A. Cowan, Andres Moreno‐De‐Luca, Henry Houlden, Reza Maroofian   +45 more
wiley   +1 more source

The clinical spectrum of SMA‐PME and in vitro normalization of its cellular ceramide profile

Annals of Clinical and Translational Neurology, Volume 9, Issue 12, Page 1941-1952, December 2022., 2022
Abstract Objective The objectives of this study were to define the clinical and biochemical spectrum of spinal muscular atrophy with progressive myoclonic epilepsy (SMA‐PME) and to determine if aberrant cellular ceramide accumulation could be normalized by enzyme replacement.
Michelle M. Lee, Graeme S. V. McDowell, Darryl C. De Vivo, Daniel Friedman, Samuel F. Berkovic, Maria Spanou, Argirios Dinopoulos, Katheryn Grand, Pedro A. Sanchez‐Lara, Michelle Allen‐Sharpley, Jodi Warman‐Chardon, Alexander Solyom, Thierry Levade, Edward H. Schuchman, Steffany A. L. Bennett, David A. Dyment, Toni S. Pearson   +16 more
wiley   +1 more source

Recognizing early MRI signs (or their absence) is crucial in diagnosing metachromatic leukodystrophy

Annals of Clinical and Translational Neurology, Volume 9, Issue 12, Page 1999-2009, December 2022., 2022
Abstract Objectives Metachromatic leukodystrophy (MLD) has characteristic white matter (WM) changes on brain MRI, which often trigger biochemical and genetic confirmation of the diagnosis. In early or pre‐symptomatic disease stages, these typical MRI changes might be absent, hampering early diagnosis.
Daphne H. Schoenmakers, Shanice Beerepoot, Ingeborg Krägeloh‐Mann, Saskia Elgün, Benjamin Bender, Marjo S. van der Knaap, Nicole I. Wolf, Samuel Groeschel   +7 more
wiley   +1 more source

Combining CD34+ stem cell selection with prophylactic pathogen and leukemia directed T‐cell immunotherapy to simultaneously reduce graft versus host disease, infection, and leukemia recurrence after allogeneic stem cell transplant

American Journal of Hematology, Volume 98, Issue 1, Page 159-165, January 2023., 2023
Donor stem cell product undergoes CD34 stem cell selection. CD34 positive stem cells are used for transplantation. Pathogen‐ and leukemia‐specific T‐cells are manufactured from CD34 negative fraction and infused prophylactically 21 days after transplant.
David J. Gottlieb, Gaurav Sutrave, Wei Jiang, Selmir Avdic, Janine A. Street, Renee Simms, Leighton E. Clancy, Vicki Antonenas, Brian S. Gloss, Caroline Bateman, David C. Bishop, Kenneth P. Micklethwaite, Emily Blyth   +12 more
wiley   +1 more source

Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB‐206 study

American Journal of Hematology, Volume 98, Issue 1, Page 11-22, January 2023., 2023
Treatment process evolution. *The target TNC for BMH was ≥6 × 108/kg per patient. The Group B1 patient received lovo‐cel produced using both the original and refined manufacturing process, and the Group B2 patient received lovo‐cel produced using only the refined manufacturing process.
Julie Kanter, Alexis A. Thompson, Francis J. Pierciey Jr, Matthew Hsieh, Naoya Uchida, Philippe Leboulch, Manfred Schmidt, Melissa Bonner, Ruiting Guo, Alex Miller, Jean‐Antoine Ribeil, David Davidson, Mohammed Asmal, Mark C. Walters, John F. Tisdale   +14 more
wiley   +1 more source

Cutaneous T‐cell lymphomas: 2023 update on diagnosis, risk‐stratification, and management

American Journal of Hematology, Volume 98, Issue 1, Page 193-209, January 2023., 2023
Abstract Disease Overview Cutaneous T‐cell lymphomas are a heterogenous group of T‐cell neoplasms involving the skin, the majority of which may be classified as Mycosis Fungoides (MF) or Sézary Syndrome (SS). Diagnosis The diagnosis of MF or SS requires the integration of clinical and histopathologic data.
Alexandra C. Hristov, Trilokraj Tejasvi, Ryan A. Wilcox   +2 more
wiley   +1 more source

Compassionate use of ruxolitinib in acute and chronic graft versus host disease refractory both to corticosteroids and extracorporeal photopheresis

Experimental Hematology & Oncology, 2017
Background Ruxolitinib is a potent inhibitor of JAK1/2 with proven efficacy in myelofibrosis. In recent years, research in graft versus host disease (GVHD) has revealed the role of activation of JAK pathways in alloreactive lymphocytes. Some reports have
Mauricio Sarmiento Maldonado, Pablo Ramírez Villanueva, Pablo Bertín Cortes-Monroy, Veronica Jara Arias, Katherine Soto Donoso, Pablo Uribe Gonzalez, Mauricio Ocqueteau Tachini, Jose Antonio Perez-Simón   +7 more
doaj   +1 more source

Single-cell heterogeneity and dynamic evolution of Ph-like acute lymphoblastic leukemia patient with novel TPR-PDGFRB fusion gene

Experimental Hematology & Oncology, 2023
Philadelphia chromosome-like acute lymphoblastic leukemia (Ph-like ALL) is a refractory and recurrent subtype of B-cell ALL enriched with kinase-activating rearrangements.
Xuehong Zhang, Zhijie Hou, Dan Huang, Furong Wang, Beibei Gao, Chengtao Zhang, Dong Zhou, Jiacheng Lou, Haina Wang, Yuan Gao, Zhijie Kang, Ying Lu, Quentin Liu, Jinsong Yan   +13 more
doaj   +1 more source

Low-intensity pulsed ultrasound promotes mesenchymal stem cell transplantation-based articular cartilage regeneration via inhibiting the TNF signaling pathway [PDF]

Stem Cell Res Ther 14, 93 (2023), 2021
Background: Mesenchymal stem cell (MSC) transplantation therapy is highly investigated for the regenerative repair of cartilage defects. Low-intensity pulsed ultrasound (LIPUS) has the potential to promote chondrogenic differentiation of MSCs. However, its underlying mechanism remains unclear.
arxiv   +1 more source