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Publications in Drug Discovery

587. Macrophages Transplantation in RDEB Mouse for Expression of Collagen VII Using the Scavenger Promoter and Sleeping Beauty System

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

565. Feasibility and Effectiveness of Exon 51 Skipping in Human-Like MDX Mutation

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2010English

20. Development of Lentiviral Mediated Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

674. Long Term Safety of Clinical Grade LentiGlobin Vectors in a Γ-Thalassemic and Normal Mice

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

267. Development of a New Strategy for X-CGD Gene Therapy Using a miRNA Regulated Lentiviral Vector

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

556. Comparative in Vitro Transduction Efficiency of AAV Vector Serotypes 1-9 in Different Cellular Models

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

386. rAAV2/5-Mediated Gene Transfer Is Less Efficient in the Aged Rat Brain: Examination of Α-Synuclein-Mediated Neurodegeneration in the Nigrostriatal System

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

210. IPSC-Derived Hematopoietic Progenitor Cells Generated From a Murine Model of Hereditary Pulmonary Alveolar Proteinosis Recapitulate the GM-CSF Dependent Functional Defects of the Disease

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

690. Optimisation of the 2A Co-Expression System for Gene Therapies

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

622. High-Resolution, Overlapping Deposition of Multiple Plasmid DNA Species on a Cell Culture Substrate for Patterned Electroporation to a Cell Monolayer

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

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