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