Results 31 to 40 of about 1,055,467 (319)

Heterogeneity in the Global Practice of Central Nervous System Staging in Pediatric Acute Lymphoblastic Leukemia

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Central nervous system (CNS) involvement in childhood acute lymphoblastic leukemia (ALL) is assessed by cell counting and cytomorphology from cerebrospinal fluid (CSF) and is used for treatment stratification worldwide. The ratio of “CNS2” patients in clinical trials ranges from 3% to 40%, with unclear prognostic significance ...
Laura Almási, Bernadett Borbála Huszti, Gábor Barna, Marko Kavcic, Janez Jazbec, Lennart Lenk, Vera Münch, Sophia Polychronopoulou, Ágnes Márk, Maria Thastrup, Lüder H. Meyer, Kjeld Schmiegelow, Cornelia Eckert, Christina Halsey, Dániel J. Erdélyi   +14 more
wiley   +1 more source

AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models

Nature Communications
Hypertrophic cardiomyopathy (HCM) affects approximately 600,000 people in the United States. Loss-of-function mutations in Myosin Binding Protein C3, MYBPC3, are the most common genetic cause of HCM, with the majority of mutations resulting in ...
Amara Greer-Short, Anna Greenwood, Elena C. Leon, Tawny Neal Qureshi, Konor von Kraut, Justin Wong, Jonathan H. Tsui, Christopher A. Reid, Ze Cheng, Emilee Easter, Jin Yang, Jaclyn Ho, Stephanie Steltzer, Ana Budan, Marie Cho, Rishikesan Chandrakumar, Olga Cisne-Thompson, Charles Feathers, Tae Won Chung, Neshel Rodriguez, Samantha Jones, Chris Alleyne-Levy, Jun Liu, Frank Jing, William S. Prince, JianMin Lin, Kathryn N. Ivey, Whittemore G. Tingley, Timothy Hoey, Laura M. Lombardi   +29 more
doaj   +1 more source

Retinoblastoma: A review of the molecular basis of tumor development and its clinical correlation in shaping future targeted treatment strategies

Indian Journal of Ophthalmology, 2023
Retinoblastoma is a retinal cancer that affects children and is the most prevalent intraocular tumor worldwide. Despite tremendous breakthroughs in our understanding of the fundamental mechanisms that regulate progression of retinoblastoma, the ...
Shruti Rathore, Aman Verma, Ria Ratna, Navjot Marwa, Yagya Ghiya, Santosh G Honavar, Anil Tiwari, Sima Das, Akhil Varshney   +8 more
doaj   +1 more source

THERAPEUTIONS

The American Journal of the Medical Sciences, 1892
n ...
openaire   +2 more sources

The Role of Hematopoietic Cell Transplantation in Ataxia‐Telangiectasia

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Ataxia‐telangiectasia (A‐T) is a DNA repair disorder characterized by neurodegeneration, immunodeficiency, and cancer predisposition. Hematopoietic cell transplantation (HCT) is an established therapy in related disorders such as Fanconi anemia (FA) and Nijmegen breakage syndrome (NBS), but its role in A‐T is unclear.
Laila Alkhouli, Seth Rotz, Masatoshi Takagi, Richa Sharma   +3 more
wiley   +1 more source

WRN inhibition leads to its chromatin-associated degradation via the PIAS4-RNF4-p97/VCP axis

Nature Communications
Synthetic lethality provides an attractive strategy for developing targeted cancer therapies. For example, cancer cells with high levels of microsatellite instability (MSI-H) are dependent on the Werner (WRN) helicase for survival.
Fernando Rodríguez Pérez, Dean Natwick, Lauren Schiff, David McSwiggen, Alec Heckert, Melina Huey, Huntly Morrison, Mandy Loo, Rafael G. Miranda, John Filbin, Jose Ortega, Kayla Van Buren, Danny Murnock, Arnold Tao, Renee Butler, Kylie Cheng, William Tarvestad, Zhengjian Zhang, Eric Gonzalez, Rand M. Miller, Marcus Kelly, Yangzhong Tang, Jaclyn Ho, Daniel Anderson, Charlene Bashore, Stephen Basham   +25 more
doaj   +1 more source

A Revolution in Cellular Aging: A Narrative Review of the Promising Role of Nanorobots in Diagnosis, Treatment, and Regenerative Medicine [PDF]

Taṣvīr-i salāmat
Nanorobots, also known as nanobots, are promising medical structures with significant potential for revolutionizing medical treatments and preventing their onset. Nanorobotics shows promise for diagnosing, treating, and regenerating age-related diseases.
Peyman Keyhanvar, Mohammad Hossein Rezaei, Hakimeh Hazrati, Solmaz Hazratgholizad, Ahmadreza Safar Bakhshayesh   +4 more
doaj   +1 more source

Framework nucleic acids directed assembly of Au nanostructures for biomedical applications

Interdisciplinary Medicine, 2023
Au nanostructures have elicited considerable interest because of their unique optical and physicochemical properties. These characteristics are mostly depended on the size, shape, chemical composition and dielectric environment of Au nanoparticles (AuNPs)
Bing Liu, Zhonglin Qi, Jie Chao
doaj   +1 more source

Therapeutic Apheresis for Intravenous Methylprednisolone‐Refractory Neuromyelitis Optica Spectrum Disorder: Clinical and Radiological Outcomes in a Single‐Center Case Series

Therapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Background Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing autoimmune disease of the central nervous system. High‐dose intravenous methylprednisolone (IVMP) is the standard first‐line therapy for acute attacks, although some patients remain refractory.
Wataru Horiguchi, Daisuke Katagiri, Takeyuki Watadani, Shunichi Matsuda, Hiroshi Kaneko, Noritoshi Arai   +5 more
wiley   +1 more source

Characterization of exoribonuclease XRN1 as a cancer target and identification of adenosine-3’,5’-bisphosphate as a potent enzyme inhibitor

Communications Biology
XRN1 (5’-3’ exoribonuclease 1) degrades RNA from the 5′ → 3′ direction and utilizes both single- and double-stranded RNA as substrates. XRN1 plays a critical role in mRNA turnover as well as regulating the cellular response to viral infection.
Gordon J. Lockbaum, Maureen M. Lynes, Sophie A. Shen, Julie Liu, Nicholas Holt, Sunaina P. Nayak, Kevin E. Knockenhauer, Shihua Yao, E. Allen Sickmier, Anugraha Raman, Jie Wu, April Case, Livia Shehaj, Shane M. Buker, Simina Grigoriu, Scott Ribich, Stephen J. Blakemore, Brian A. Sparling, Kenneth W. Duncan, Robert A. Copeland, Serena J. Silver, P. Ann Boriack-Sjodin   +21 more
doaj   +1 more source