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Advance in the Treatment of Pediatric Leukemia
| Advance in the Treatment of Pediatric Leukemia |
| Autore | Handgretinger Rupert |
| Pubbl/distr/stampa | Basel, : MDPI - Multidisciplinary Digital Publishing Institute, 2022 |
| Descrizione fisica | 1 online resource (300 p.) |
| Soggetto topico |
Chemistry
Research & information: general |
| Soggetto non controllato |
5-azacitidine
ABCC4 acute lymphoblastic leukemia acute myeloid leukemia acute pancreatitis ADC advances ALL allogeneic stem cell transplantation alternative donors AML antibody antibody-drug conjugate B-ALL BCL11B BCP-ALL bispecific T-cell engager (BiTE) CD19 CFTR childhood acute lymphoblastic leukemia children chronic myeloid leukemia CML conditioning regimen COVID-19 diagnosis DUX4 evolution of CAR T cells Fc-engineering FDA-approved CAR products future directions of CAR T cell therapy genome hematopoietic stem cell transplantation IKZF1 immunizations immunotherapy juvenile myelomonocytic leukemia L-asparaginase leukemia limitations and complications of CAR T cell therapy low-risk ALL lymphoblastic leukemia minimal residual disease myelodysplastic/myeloproliferative disorders n/a NOTCH1 NUTM1 other extramedullary relapse PAX5 pediatric pediatric leukemia Ph-like polymorphism prognosis RAS signaling risk-stratified treatment SNV T-ALL targeted therapy TcR versus CAR TRAIL transcriptome treatment treatment related toxicity tyrosine kinase inhibitor xenograft ZNF384 |
| Formato | Materiale a stampa  |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Record Nr. | UNINA-9910576878903321 |
Handgretinger Rupert
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| Basel, : MDPI - Multidisciplinary Digital Publishing Institute, 2022 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function
| Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function |
| Autore | Duddy William |
| Pubbl/distr/stampa | Basel, Switzerland, : MDPI - Multidisciplinary Digital Publishing Institute, 2021 |
| Descrizione fisica | 1 online resource (318 p.) |
| Soggetto topico | Research & information: general |
| Soggetto non controllato |
-omics approaches
AAV adult patients ALS ALS genes ALS pathology ALS variants Amyotrophic Lateral Sclerosis autophagy axonal transport Becker muscular dystrophy becker muscular dystrophy (BMD) biomarkers calprotectin Canadian Neuromuscular Disease Registry clinical trials comparative genomic hybridization array (CGH) corticosteroids CRISPR-Cas9 D4Z4 deflazacort disease heterogeneity disease models disease modifiers DMARD DMD Duchenne muscular dystrophy duchenne muscular dystrophy (DMD) Duchenne muscular dystrophy (DMD) DUX4 dystrophin dystrophinopathy dystrophy Emery-Dreifuss muscular dystrophy endocytosis epigenetic changes eteplirsen excitotoxicity exon skipping exon skipping therapy exon-skipping therapies facioscapulohumeral dystrophy FSHD functional outcomes gene editing gene prioritization gene therapy genetic neuromuscular disorders genome-wide association studies genomics genotype-phenotype genotype-phenotype correlations glucocorticoids golodirsen GWAS LMNA machine learning methotrexate miRNA mitochondria dysfunction MND multiple logistic regression analysis multiplex ligation probe amplification (MLPA) multiplex polymerase chain reaction (PCR) muscle n/a neuromuscular diseases next-generation sequencing (NGS) Nusinersen Omics oxidative stress pharmacodynamic biomarkers pharmacogenomics polyadenylation precision medicine prednisone proteomics reading frame rule regulation rheumatoid arthritis RNA metabolism safety Sanger sequencing secretion skip-equivalent deletions SMA SNP spinal muscular atrophy TALEN toxicity transcription translational research viltolarsen XLMTM |
| Formato | Materiale a stampa |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Altri titoli varianti | Understanding Neuromuscular Health and Disease |
| Record Nr. | UNINA-9910557669103321 |
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Duddy William
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| Basel, Switzerland, : MDPI - Multidisciplinary Digital Publishing Institute, 2021 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
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