Results 191 to 200 of about 185,548 (298)

Incidence, clinical features and risk factors of interstitial pneumonia in children with acute lymphoblastic leukemia. [PDF]

Ann Hematol
Li Y, Fan Z, Wang LN, Tang YL, Zhang XL, Tan HZ, Zhang YC, Liang C, Liu XJ, Guo CY, Luo XQ, Huang LB, Ke ZY.   +12 more
europepmc   +1 more source

A new recurrent inversion, inv(7)(p15q34), leads to transcriptional activation of HOXA10 and HOXA11 in a subset of T-cell acute lymphoblastic leukemias [PDF]

, 2005
Benoit, Yves, BILLIET, J, BOOGAERTS, M, Cauwelier, Barbara, COOLS, J, DASTUGUE, N, De Moerloose, Barbara, De Paepe, Anne, GRAUX, C, HAGEMEIJER, A, HUGUET, F, MARYNEN, P, Poppe, Bruce, ROBERT, A, SELLESLAG, D, Speleman, Franki, UYTTEBROECK, A, Van Roy, Nadine, VANDENBERGHE, P, Vandesompele, Jo, Verhasselt, Bruno   +20 more
core  

Dimerization‐dependent NOTCH receptor transactivation unveils a class of highly selective NOTCH signalling inhibitors

The FEBS Journal, EarlyView.
NOTCH signalling is indispensable for tissue homeostasis and, consequently, corruption of its normal function promotes numerous diseases, including cancer. However, the development of targeted therapies has been hampered by inefficacy and overt toxicity. Here, we show that NOTCH receptor dimerization is necessary for receptor transactivation, which has
Xinxin Liu, Haijiang Wang, Gunja Mishra, Lin‐Ting Wu, Chao Li, Maarten van Dinther, Jin Liu, Manuel A.F.V. Gonçalves, Peter ten Dijke, David A. Baker   +9 more
wiley   +1 more source

Multi-Omics Applications in Adult Acute Lymphoblastic Leukemia: From Biological Mechanisms to Precision Therapies. [PDF]

Int J Mol Sci
Simio C, Molica M, De Fazio L, Rossi M.
europepmc   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

Identification of the Philadelphia-like subgroup in Turkish pediatric patients with acute lymphoblastic leukemia. [PDF]

Ann Hematol
Efendi Erdem E, Cecener G, Unal U, Tezcan Unlu H, Sezgin Evim M, Baytan B, Egeli U, Oymak Y, Tunca B, Gunes AM.   +9 more
europepmc   +1 more source

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5 (AAV5)‐mediated gene therapy for severe haemophilia A (HA). Aim Report the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec. Methods An open‐label phase 1/2 trial (NCT02576795) enrolled adult males with severe HA (factor
Priyanka Raheja, Savita Rangarajan, Will Lester, Bella Madan, Glenn F. Pierce, Emily Symington, Carolyn Millar, Dane Osmond, Mingjin Li, Konstantia‐Maria Chavele   +9 more
wiley   +1 more source

SETD4 as a marker of disease burden and treatment response in childhood acute lymphoblastic leukemia<i>SETD4</i> as a marker of disease burden and treatment response in childhood acute lymphoblastic leukemia. [PDF]

Oncol Lett
Telles LAM, De Loyola MB, Sakamoto LHT, Rabello DDAR, Motoyama AB, Pittella-Silva F.   +5 more
europepmc   +1 more source

Clinical and Genetic Characterization of 269 Patients With Suspected Inherited Platelet Disorders: The Padua Monocentric Experience

International Journal of Laboratory Hematology, EarlyView.
ABSTRACT Background Inherited platelet disorders (IPDs) are rare hematologic conditions encompassing a heterogeneous spectrum of quantitative and qualitative platelet defects, frequently associated with variable clinical phenotypes and comorbidities. Accurate diagnosis necessitates comprehensive genetic characterization, detailed clinical and bleeding ...
Silvia Ferrari, Daniela Regazzo, Antonella Bertomoro, Anna Cerbo, Alessandro Galbiati, Elisabetta Cosi, Paolo Simioni   +6 more
wiley   +1 more source