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Publications in Drug Discovery

618. Adjunctive Γ2-Agonist Therapy Enhances Trafficking of Acid Α-Glucosidase to Lysosomes in the Skeletal Muscle of Mice With Pompe Disease

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

147. Abstract Withdrawn

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

Improving mRNA-based Therapeutic Gene Delivery by Expression Augmenting 3’-Untranslated Regions Identified by Cellular Library Screening

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2019English

287. Improved Outcomes Following Drug-Resistant Immunotherapy in a Human Xenograft Model of Temozolomide-Resistant Glioblastoma Multiforme

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

342. Utilizing Shielded Sliding Plasma Discharges Generated in Ambient Air for Delivery of Plasmid DNA to Mammalian Cells

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

Long-Term Correction of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

84. Generation of Functional Monocyte/Macrophages by Genetic Correction of Patient-Specific iPSC in Congenital Pulmonary Alveolar Proteinosis

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

604. Intrathecal AAV9-Mediated Gene Delivery Corrects Lysosomal Storage Throughout the CNS in a Large Animal Model of Mucopolysaccharidosis Type I

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

Phase 1 Study of Intravenous Oncolytic Poxvirus (vvDD) in Patients With Advanced Solid Cancers

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

The Other Face of Chimeric Antigen Receptors

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

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