Accès ouvert
2026
article
OpenAlex
Andrea Timpanaro, Edward Z. Song, Ryma Toumi, Leonel Elena-Sanchez et autres
Background Diffuse intrinsic pontine glioma (DIPG) is a fatal pediatric brain tumor affecting over 300 children annually in the United States. Chimeric antigen receptor (CAR) T cells are a targeted immune effector cell therapy with substantial clinical benefit against hematologic cancers. Against …
Accès ouvert
2026
article
OpenAlex
Andrea Timpanaro, Edward Z. Song, Ryma Toumi, Leonel Elena-Sanchez et autres
BACKGROUND: Diffuse intrinsic pontine glioma (DIPG) is a fatal pediatric brain tumor affecting over 300 children annually in the United States. Chimeric antigen receptor (CAR) T cells are a targeted immune effector cell therapy with substantial clinical benefit against hematologic cancers. Against …
us, au
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Accès ouvert
2025
article
OpenAlex
Edward Z. Song, Andrea Timpanaro, Michael Meechan, Leonel Elena-Sanchez et autres
Diffuse intrinsic pontine glioma (DIPG) is a fatal brainstem tumor desperately in need of better treatments. Chimeric antigen receptor (CAR) T cell therapies for DIPG have demonstrated clinical tolerability and bioactivity, but not universal benefit. A major obstacle is insufficient CAR T …
us, au
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Accès ouvert
2025
article
OpenAlex
Randolph K. Larsen, Jason A. Hanna, Hongjian Jin, Kristin B. Reed et autres
DICER1-related tumors are characterized by germline loss-of-function mutations in one DICER1 allele ( DICER1 +/− ) and a somatic “second hit” mutation in the remaining DICER1 allele. Whether the germline DICER1 +/− mutation participates in tumorigenesis is unknown. We show that germline …
us
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Accès ouvert
2025
article
OpenAlex
Katherine E. Gadek, Catherine J. Drummond, Hongjian Jin, Matthew R. Garcia et autres
Rhabdomyosarcoma (RMS) is a pediatric soft tissue sarcoma resembling developing skeletal muscle, but tumors are found in sites devoid of skeletal muscle, suggesting a non-myogenic cell of origin. We previously described an endothelial progenitor cell-derived model of RMS by activating a conditional, …
us
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Accès ouvert
2025
preprint
OpenAlex
Andrea Timpanaro, Edward Z. Song, Ryma Toumi, Leonel Elena-Sanchez et autres
ABSTRACT Background Diffuse intrinsic pontine glioma (DIPG) is a fatal pediatric brain tumor affecting over 300 children annually in the United States. Chimeric antigen receptor (CAR) T cells are a targeted immune effector cell therapy with substantial clinical benefit against hematologic cancers. …
us, au
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Accès ouvert
2025
conference-abstract
OpenAlex
Isabella Terrones, Hawa Jagana, Matthew Hathaway, John Hemenway et autres
Abstract Medulloblastoma (MB) is the most common malignant pediatric brain tumor with Group 3 (G3) tumors having the lowest overall survival rate. Using the Pediatric Cancer Dependency Map to screen for therapeutic dependencies, we previously identified cyclin-dependent kinase 8 (CDK8) as the …
us
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Accès ouvert
2025
conference-abstract
OpenAlex
Stephen V. Carney, John Hemenway, Kate E. Gadek, Christian Bonatto Paese et autres
Abstract Current standard of care treatment for medulloblastoma (MB) consists of maximal resection surgery followed by craniospinal irradiation and chemotherapy, with survivors experiencing diminished quality of life from intensive treatment regimens. The identification of novel targets is an unmet need and critical …
us
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Accès ouvert
2025
article
OpenAlex
Lena M. Kutscher, Davide Aprile, Nermin Sumru Bayin, Esther B. E. Becker et autres
Acknowledgements: We thank all participants for their contributions. We thank the sponsors for the meeting for financial and logistical support.
de, it, gb, fr, us, ca
(code pays fourni par la source)
2025
conference-abstract
OpenAlex
Andrea Timpanaro, Edward Z. Song, Matthew C. Biery, Ryma Toumi et autres
B7-H3 CAR T cell treatment in combination with ONC206 aims to assess benefit and significant extension of survival in orthotopic DIPG mouse models. BACKGROUND. Diffuse intrinsic pontine glioma (DIPG) is a fatal pediatric tumor arising in the pons and affecting over 300 …
us, au
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Accès ouvert
2025
preprint
OpenAlex
Nicholas Alexander Vitanza, Edward Z. Song, Andrea Timpanaro, Michael Meechan et autres
us
(code pays fourni par la source)
2024
conference-abstract
OpenAlex
Randolph K. Larsen, Jason A. Hanna, Hongjian Jin, Darden W. Kimbrough et autres
Abstract DICER1 cancer predisposition syndrome is a heritable condition where affected patients have increased risk of neoplasms including fusion-negative rhabdomyosarcoma (FN-RMS). The syndrome is defined by germline heterozygous loss of function mutations in DICER1, but it remains unclear exactly how these mutations …
us
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