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Publications in Drug Discovery

462. Engineering the Human Factor VIII Signal Peptide for Enhanced Secretion

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

514. Robust RNAi Enhancement via Human Argonaute-2 Overexpression From Plasmids, Viral Vectors or Cell Lines

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

264. Translating RNAi Therapy for Spinocerebellar Ataxia 1 (SCA1) to the Clinic

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

543. Creation of Structural Variations in the Human Genome Using Engineered Zinc Finger Nucleases

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

212. Disease Modeling of Late-Onset Pompe Disease-Specific Induced Pluripotent Stem Cells

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

Dystrophin Isoform Induction in Vivo by Antisense-Mediated Alternative Splicing

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2010English

[18F]FHBG PET/CT Imaging of CD34-TK75 Transduced Donor T Cells in Relapsed Allogeneic Stem Cell Transplant Patients: Safety and Feasibility

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

Taking a Hint From Structural Biology: To Better Understand AAV Transport Across the BBB

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2018English

680. Gene Therapy of Chondroitin Sulfate-Coated Ternary GM-CSF Plasmid Complex for Ovarian Cancer

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

Mesenchymal Stem Cells Correct Inappropriate Epithelial–mesenchyme Relation in Pulmonary Fibrosis Using Stanniocalcin-1

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

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