Results 201 to 210 of about 124,423 (315)

Metachronous B-Cell Acute Lymphoblastic Leukemia After Localized Neuroblastoma Treated Without Cytotoxic Therapy: A Case Report. [PDF]

Case Rep Hematol
Sedghi R, Nazar Zadeh A, Bordbar M, Zekavat OR, Parand S.   +4 more
europepmc   +1 more source

A quantitative dissection of the DNA‐binding properties of pathogenic GATA1 mutants

The FEBS Journal, EarlyView.
Transcription factor activity is influenced by cell context, accessibility of target sequences, and co‐factor recruitment. Quantitatively characterizing the consequences of individual mutations in the transcription factors or their target sequences remains technically challenging. Zambo et al. use mutant GATA1‐ATP2B4 binding to illustrate an innovation
Kaoru Takasaki
wiley   +1 more source

Adding induction intrathecal cytarabine in newly diagnosed CNS2 B-acute lymphoblastic leukemia does not improve outcomes. [PDF]

Blood Adv
Lee-Miller CA, Kairalla JA, Hibbitts E, Winick NJ, Rabin KR, Angiolillo A, Schore RJ, Salzer WL, Burke MJ, Loh ML, Raetz EA, Hunger SP, Devidas M, Teachey DT, Gupta S, McNeer JL.   +15 more
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

Association of neuropathy, sarcopenia and physical function in acute lymphoblastic leukemia survivors with chemotherapy alone. [PDF]

J Cancer Surviv
Fridh MK, Xing M, Mirzaei S, Mizrahi D, Larsen HB, Dixon SB, Phillips NS, Krull KR, Inaba H, Pui CH, Karol SE, Delaney A, Kaste SC, Hudson MM, Ness KK.   +14 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

Philadelphia Chromosome-Positive B-cell Acute Lymphoblastic Leukemia: A Case Report. [PDF]

Cureus
Izadian Bidgoli A, Gomez Veliz A, Yazdanpanah S, Saavedra G, Pina A, Rame G.   +5 more
europepmc   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source

Severe Thiopurine-Induced Myelosuppression in a Pediatric Acute Lymphoblastic Leukemia Patient With the NUDT15 *1/*6 Genotype: A Brief Report. [PDF]

Clin Transl Sci
Fry J, Boone EC, Wang WY, John S, Lansdon LA, Farooqi MS, Yoo B, Gaedigk A, August K, Flatt TG, Ramsey LB.   +10 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