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Publications in Drug Discovery

554. CRISPR/Cas9-Based Transcriptional Activation of Endogenous Myod1 to Reprogram Murine Fibroblasts Into Skeletal Myocytes

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

579. Complete Tropism Restriction by Display of High Affinity Ligands on Adeno-Associated Viral Vectors

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

726. Retroviral Vector Gene Therapy Delays Mitral Valve Regurgitation in Mucopolysaccharidosis VII Dogs but Does Not Prevent Disease Long Term Due to Failure to Completely Prevent Induction of Inflammaory Pathways

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

264. Absence of CD8 T Cell Responses Against AAV Capsid Using Reduced Vector Dose or Library Selected Liver Targeted AAV2 Based Capsids

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

523. Reducing in Vivo Clearance of Lentiviral Vectors by Immune Cells in Order to Improve Efficacy and Safety of Gene Therapy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

377. Significant Therapeutic Benefit After Post-Symptomatic Gene Therapy in a Feline Model of GM2 Gangliosidosis

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

177. Development of Peptide-Based HPMA-Copolymers for Nucleic Acid Delivery

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

122. Seamless Correction by Donor DNA of a Class I CFTR Mutation Facilitated by a Double Nicking CRISPR/Cas9 in CF-iPSCs

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

857. Donor Dependency of Integrin-And Chemokine Mediated Adhesion Activation in Human Mesenchymal Stromal Cells (MSCs)

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

203. Phase I Study of the “Triad” Autologous (FANG™) Vaccine, Incorporating Bifunctional shRNAfurin and GMCSF Transgene Expression, in Advanced Ewing's Sarcoma: Preliminary Data in Pediatrics Patients

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

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