Result Number | Material Type | Add to My Shelf Action | Record Details and Options |
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1 |
Material Type: Other
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Inflammatory Myofibroblastic Tumor of the Abdomen: Clinicopathological and Molecular Characterization of Seventeen Cases, Including Three Epithelioid Inflammatory Myofibroblastic Sarcomas, Highlighting Potential Therapeutic Targetspublic 720193;All rights reserved ;ISSN: 1530-0307 ;ISSN: 0023-6837 ;EISSN: 1530-0307Full text available |
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2 |
Material Type: Other
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UNRAVELLING DRUG RESISTANCE AND SENSITIZATION TO PROTEASOME INHIBITORS USING GENOME-WIDE CRISPR-CAS9 KNOCKOUT SCREENINGintranet 731067;All rights reserved ;ISSN: 1873-2399 ;ISSN: 0301-472X ;EISSN: 1873-2399Digital Resources/Online E-Resources |
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3 |
Material Type: Other
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Building a Genetic Medicine Platform for DNA-Encoded Antibody Therapeuticsintranet 666484 ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024Full text available |
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4 |
Material Type: Other
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Intraparenchymal Convection Enhanced Delivery of AAV in Sheep to Treat Mucopolysaccharidosis IIICpublic 742025;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024Digital Resources/Online E-Resources |
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5 |
Material Type: Other
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Identification of Universal Parameters That Play a Critical Role in rAAV Production Using a Transient Two-Plasmid Packaging System in the Adherent HEK293T Cell Linepublic 745164;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024Digital Resources/Online E-Resources |
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6 |
Material Type: Other
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Development of a ddPCR Protocol for the Quantification of AAV Viral Genomes in Purified Samplespublic 742226;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024Digital Resources/Online E-Resources |
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7 |
Material Type: Other
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Gene Therapy for Adenosine Deaminase Deficiency: Long-Term Outcome and Post-Marketing Experienceintranet 745435;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024Digital Resources/Online E-Resources |
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8 |
Material Type: Other
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Long-term catch-up growth in children with acid sphingomyelinase deficiency treated with olipudase adlfa enzyme replacement therapy in the ASCEND-Peds trialintranet 727208 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2022.107115Digital Resources/Online E-Resources |
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9 |
Material Type: Other
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Base editing strategy to repair the CFTR 2789+5G > A splicing mutationintranet 703507;All rights reserved ;ISSN: 1557-7422 ;ISSN: 1043-0342 ;EISSN: 1557-7422Full text available |
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10 |
Material Type: Other
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Nanoblades allow high-level genome editing in organoidsintranet 703493;All rights reserved ;ISSN: 1557-7422 ;ISSN: 1043-0342 ;EISSN: 1557-7422Full text available |
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11 |
Material Type: Other
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A participatory framework for plain language clinical management guideline developmentpublic 728231;https://creativecommons.org/licenses/by/4.0 ;ISSN: 0300-7995 ;ISSN: 1473-4877 ;EISSN: 1473-4877Digital Resources/Online E-Resources |
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12 |
Material Type: Other
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Biodistribution and Comparative Performance of AAV-TT and AAV9 in the Nonhuman Primate Brainintranet 671227 ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024Full text available |
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13 |
Material Type: Other
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CLINICAL AND BIOCHEMICAL FOOTPRINTS OF INHERITED METABOLIC DISORDERS: A LESSON FROM THE KNOWLEDGEBASEpublic 660361;All rights reserved ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206Digital Resources/Online E-Resources |
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14 |
Material Type: Other
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Continued improvement in pulmonary, visceral, biomarker and growth outcomes in children with chronic acid sphingomyelinase deficiency treated with olipudase alfa enzyme replacement therapy: 2-year results of ASCEND-Pedsintranet 680206 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2021.11.082Digital Resources/Online E-Resources |
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15 |
Material Type: Other
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Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trialsintranet 676998 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2021.11.058Digital Resources/Online E-Resources |
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16 |
Material Type: Other
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Continued improvement in pulmonary outcomes in 3 clinical trials of olipudase alfa in children and adults with chronic acid sphingomyelinase deficiency treated for 2 to 6.5 yearsintranet 681950;All rights reserved ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2021.11.285Digital Resources/Online E-Resources |
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17 |
Material Type: Other
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THE METABOLIC MAP OF THE PATHOMECHANISM AND TREATMENT OF PGM1-CDGintranet 595153 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206Digital Resources/Online E-Resources |
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18 |
Material Type: Other
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Inhibiting RIPK1 Kinase Activity Is Protective in Experimental Models of COPDpublic 676792;All rights reserved ;ISSN: 1073-449X ;ISSN: 1535-4970 ;EISSN: 1535-4970Full text available |
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19 |
Material Type: Other
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Overexpression of Catalase in Mitochondria Mitigates the Effects of Simulated Microgravity and Social Isolation on Cytokine Expression in Mouse HippocampusCopyright Determination: PUBLIC_USE_PERMITTEDDigital Resources/Online E-Resources |
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20 |
Material Type: Other
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Implementation of Model-Based Design of Experiments: Application of Computational Modeling to Support HRP StudiesCopyright Determination: PUBLIC_USE_PERMITTEDDigital Resources/Online E-Resources |