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1
Inflammatory Myofibroblastic Tumor of the Abdomen: Clinicopathological and Molecular Characterization of Seventeen Cases, Including Three Epithelioid Inflammatory Myofibroblastic Sarcomas, Highlighting Potential Therapeutic Targets
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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 Targets

public 720193;All rights reserved ;ISSN: 1530-0307 ;ISSN: 0023-6837 ;EISSN: 1530-0307

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2
UNRAVELLING DRUG RESISTANCE AND SENSITIZATION TO PROTEASOME INHIBITORS USING GENOME-WIDE CRISPR-CAS9 KNOCKOUT SCREENING
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UNRAVELLING DRUG RESISTANCE AND SENSITIZATION TO PROTEASOME INHIBITORS USING GENOME-WIDE CRISPR-CAS9 KNOCKOUT SCREENING

intranet 731067;All rights reserved ;ISSN: 1873-2399 ;ISSN: 0301-472X ;EISSN: 1873-2399

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3
Building a Genetic Medicine Platform for DNA-Encoded Antibody Therapeutics
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Building a Genetic Medicine Platform for DNA-Encoded Antibody Therapeutics

intranet 666484 ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024

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4
Intraparenchymal Convection Enhanced Delivery of AAV in Sheep to Treat Mucopolysaccharidosis IIIC
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Intraparenchymal Convection Enhanced Delivery of AAV in Sheep to Treat Mucopolysaccharidosis IIIC

public 742025;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024

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5
Identification of Universal Parameters That Play a Critical Role in rAAV Production Using a Transient Two-Plasmid Packaging System in the Adherent HEK293T Cell Line
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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 Line

public 745164;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024

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6
Development of a ddPCR Protocol for the Quantification of AAV Viral Genomes in Purified Samples
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Development of a ddPCR Protocol for the Quantification of AAV Viral Genomes in Purified Samples

public 742226;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024

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7
Gene Therapy for Adenosine Deaminase Deficiency: Long-Term Outcome and Post-Marketing Experience
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Gene Therapy for Adenosine Deaminase Deficiency: Long-Term Outcome and Post-Marketing Experience

intranet 745435;All rights reserved ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024

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8
Long-term catch-up growth in children with acid sphingomyelinase deficiency treated with olipudase adlfa enzyme replacement therapy in the ASCEND-Peds trial
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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 trial

intranet 727208 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2022.107115

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9
Base editing strategy to repair the CFTR 2789+5G > A splicing mutation
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Base editing strategy to repair the CFTR 2789+5G > A splicing mutation

intranet 703507;All rights reserved ;ISSN: 1557-7422 ;ISSN: 1043-0342 ;EISSN: 1557-7422

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10
Nanoblades allow high-level genome editing in organoids
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Nanoblades allow high-level genome editing in organoids

intranet 703493;All rights reserved ;ISSN: 1557-7422 ;ISSN: 1043-0342 ;EISSN: 1557-7422

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11
A participatory framework for plain language clinical management guideline development
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A participatory framework for plain language clinical management guideline development

public 728231;https://creativecommons.org/licenses/by/4.0 ;ISSN: 0300-7995 ;ISSN: 1473-4877 ;EISSN: 1473-4877

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12
Biodistribution and Comparative Performance of AAV-TT and AAV9 in the Nonhuman Primate Brain
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Biodistribution and Comparative Performance of AAV-TT and AAV9 in the Nonhuman Primate Brain

intranet 671227 ;ISSN: 1525-0024 ;ISSN: 1525-0016 ;EISSN: 1525-0024

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13
CLINICAL AND BIOCHEMICAL FOOTPRINTS OF INHERITED METABOLIC DISORDERS: A LESSON FROM THE KNOWLEDGEBASE
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CLINICAL AND BIOCHEMICAL FOOTPRINTS OF INHERITED METABOLIC DISORDERS: A LESSON FROM THE KNOWLEDGEBASE

public 660361;All rights reserved ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206

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14
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-Peds
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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-Peds

intranet 680206 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2021.11.082

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15
Overlapping and divergent hepatic and lipoprotein phenotypes in untreated adults with acid sphingomyelinase deficiency versus untreated adults with Gaucher disease from two pivotal clinical trials
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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 trials

intranet 676998 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2021.11.058

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16
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 years
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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 years

intranet 681950;All rights reserved ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206 ;DOI: 10.1016/j.ymgme.2021.11.285

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17
THE METABOLIC MAP OF THE PATHOMECHANISM AND TREATMENT OF PGM1-CDG
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THE METABOLIC MAP OF THE PATHOMECHANISM AND TREATMENT OF PGM1-CDG

intranet 595153 ;ISSN: 1096-7192 ;ISSN: 1096-7206 ;EISSN: 1096-7206

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18
Inhibiting RIPK1 Kinase Activity Is Protective in Experimental Models of COPD
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Inhibiting RIPK1 Kinase Activity Is Protective in Experimental Models of COPD

public 676792;All rights reserved ;ISSN: 1073-449X ;ISSN: 1535-4970 ;EISSN: 1535-4970

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19
Overexpression of Catalase in Mitochondria Mitigates the Effects of Simulated Microgravity and Social Isolation on Cytokine Expression in Mouse Hippocampus
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Overexpression of Catalase in Mitochondria Mitigates the Effects of Simulated Microgravity and Social Isolation on Cytokine Expression in Mouse Hippocampus

Copyright Determination: PUBLIC_USE_PERMITTED

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20
Implementation of Model-Based Design of Experiments: Application of Computational Modeling to Support HRP Studies
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Implementation of Model-Based Design of Experiments: Application of Computational Modeling to Support HRP Studies

Copyright Determination: PUBLIC_USE_PERMITTED

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Results 1 - 20 of 93  for All Library Resources

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