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View article: High-throughput 3D engineered paediatric tumour models for precision medicine
High-throughput 3D engineered paediatric tumour models for precision medicine Open
Precision medicine for paediatric and adult cancers that incorporates drug sensitivity profiling can identify effective therapies for individual patients. However, obtaining adequate biopsy samples for high-throughput (HTP) screening remai…
View article: Table S1 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Table S1 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Single agent and combination doses established in the larval zebrafish PDX at 35°C for 3 to 7 days post fertilization zebrafish for 72-hour treatment.
View article: Figure 4 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Figure 4 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Larval zebrafish drug efficacy studies for patients with no available mouse PDX. A to C, Patient information, alongside larval zebrafish tumor cell numbers for each therapy for rhabdomyosarcoma zccs170 (A), gastrointestinal s…
View article: Table 2 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Table 2 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Larval zebrafish PDX development: sample source and cell viability
View article: Table S2 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Table S2 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Features of mouse PDX, larval zebrafish PDX and in vitro models
View article: Figure S1 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Figure S1 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
CONSORT flow diagram for patients included in the study and the criteria not met for those excluded
View article: Table 1 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Table 1 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Patient cohort details, including molecular targets, clinical response to therapy, and mouse PDX development
View article: Data from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Data from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Despite advances in precision medicine, 30% of high-risk pediatric cancers lack an actionable molecular target, hindering effective treatment and affecting survival outcomes. Although mouse patient-derived xenograft (PDX) models offer addi…
View article: Figure 3 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Figure 3 from Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Mouse and larval zebrafish PDX drug efficacy for nonresponsive patients. A to C, Patient information, alongside mouse PDX waterfall plot of maximum tumor regression, and larval zebrafish tumor cell numbers for each therapy for neuro…
View article: Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy
Modeling High-Risk Pediatric Cancers in Zebrafish to Inform Precision Therapy Open
Despite advances in precision medicine, 30% of high-risk pediatric cancers lack an actionable molecular target, hindering effective treatment and affecting survival outcomes. Although mouse patient-derived xenograft (PDX) models offer addi…
View article: A novel TRKB-activating internal tandem duplication characterizes a new mechanism of receptor tyrosine kinase activation
A novel TRKB-activating internal tandem duplication characterizes a new mechanism of receptor tyrosine kinase activation Open
View article: A novel TRKB-activating internal tandem duplication characterizes a new mechanism of receptor tyrosine kinase activation
A novel TRKB-activating internal tandem duplication characterizes a new mechanism of receptor tyrosine kinase activation Open
Precision medicine programs like the Zero Childhood Cancer Program perform comprehensive molecular analysis of patient tumors, enabling detection of novel structural variants that may be cryptic to standard techniques. Identification of th…
View article: Structure and function of the EDEM:PDI ERAD checkpoint complex
Structure and function of the EDEM:PDI ERAD checkpoint complex Open
The ERAD glycoprotein misfolding checkpoint complex de-mannosylates misfolded glycoproteins targeting them to retrotranslocation, ubiquitination, and proteasomal degradation. The complex comprises an Endoplasmic Reticulum-Degradation Enhan…
View article: Structure and function of the EDEM:PDI ERAD checkpoint complex
Structure and function of the EDEM:PDI ERAD checkpoint complex Open
The ERAD glycoprotein misfolding checkpoint complex de-mannosylates misfolded glycoproteins to enable retrotranslocation, ubiquitination, and proteasomal degradation. The complex comprises an Endoplasmic Reticulum-Degradation Enhancing α-M…
View article: Engineered paediatric tumours retain maintains tumour genotype and phenotype for precision medicine
Engineered paediatric tumours retain maintains tumour genotype and phenotype for precision medicine Open
Precision medicine for paediatric and adult cancers that includes drug sensitivity profiling, can identify effective therapies for individual patients. However, obtaining adequate biopsy samples for high-throughput (HTP) screening remains …
View article: DIPG-48. PRECISION GUIDED THERAPY PROVIDES CLINICAL BENEFIT IN H3K27-ALTERED DIFFUSE MIDLINE GLIOMA
DIPG-48. PRECISION GUIDED THERAPY PROVIDES CLINICAL BENEFIT IN H3K27-ALTERED DIFFUSE MIDLINE GLIOMA Open
BACKGROUND The utility of precision-guided therapeutic approaches to H3K27-altered diffused midline glioma (DMG) remains uncertain. The Australian Zero Childhood Cancer (ZERO) program combines molecular profiling (whole-genome sequencing (…
View article: Preclinical assessment of combined BCL-2 and MCL-1 inhibition in high-risk neuroblastoma
Preclinical assessment of combined BCL-2 and MCL-1 inhibition in high-risk neuroblastoma Open
Background: Neuroblastoma is the most common extracranial pediatric solid malignancy with high-risk patients showing survival rates less than 50 %. These tumors frequently escape apoptosis through upregulation of the anti-apoptotic protein…
View article: A novel MYB::PAIP1 oncogenic fusion in pediatric blastic plasmacytoid dendritic cell neoplasm (BPDCN) is dependent on BCL2 expression and is sensitive to venetoclax
A novel MYB::PAIP1 oncogenic fusion in pediatric blastic plasmacytoid dendritic cell neoplasm (BPDCN) is dependent on BCL2 expression and is sensitive to venetoclax Open
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive hematological malignancy with a median survival of 14 months (with a survival window of 6–28 months) from diagnosis.1 BPDCN is derived from the precursors of pla…
View article: Rescue of secretion of rare‐disease‐associated misfolded mutant glycoproteins in <scp> <i>UGGT1</i> </scp> knock‐out mammalian cells
Rescue of secretion of rare‐disease‐associated misfolded mutant glycoproteins in <span> <i>UGGT1</i> </span> knock‐out mammalian cells Open
Endoplasmic reticulum (ER) retention of misfolded glycoproteins is mediated by the ER‐localized eukaryotic glycoprotein secretion checkpoint, UDP‐glucose glycoprotein glucosyl‐transferase (UGGT). The enzyme recognizes a misfolded glycoprot…
View article: Supplementary Figures from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Figures from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Figures
View article: Supplementary Materials and Methods from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Materials and Methods from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Materials and Methods
View article: Supplementary Tables S1-S7 from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Tables S1-S7 from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Tables S1-S7
View article: Supplementary Figures and Figure Legends from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Figures and Figure Legends from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Figures 1-7 and Figure Legends
View article: Supplementary Figures from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Figures from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Figures
View article: Supplementary Figures and Figure Legends from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Figures and Figure Legends from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Figures 1-7 and Figure Legends
View article: Supplementary Materials and Methods from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Materials and Methods from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Materials and Methods
View article: Supplementary Tables S1-S7 from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Tables S1-S7 from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Tables S1-S7
View article: Data from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Data from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
For one-third of patients with pediatric cancer enrolled in precision medicine programs, molecular profiling does not result in a therapeutic recommendation. To identify potential strategies for treating these high-risk pediatric patients,…
View article: Supplementary Figures from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Supplementary Figures from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
Supplementary Figures
View article: Data from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer
Data from High-Throughput Drug Screening of Primary Tumor Cells Identifies Therapeutic Strategies for Treating Children with High-Risk Cancer Open
For one-third of patients with pediatric cancer enrolled in precision medicine programs, molecular profiling does not result in a therapeutic recommendation. To identify potential strategies for treating these high-risk pediatric patients,…