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Publications in Molecular Medicine

325. A Novel Utrophin-Based Genome Engineering Strategy for Duchenne Muscular Dystrophy Utilizing Recombinases and CRISPR/Cas9

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

330. Comparing Dual AAV Vector Strategy Efficiencies for Gene Therapy in Vitro and in Vivo

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

Research Highlights

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2018English

640. AdCD40L Immunostimulatory Gene Therapy in Combination With Cyclophosphamide Prolongs 6-Months Survival in a Phase I/Ii Trial for Malignant Melanoma

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

71. The Structure of AAVrh32.33, a Novel Gene Delivery Vector

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

Monocyte/Macrophage-Derived IGF-1 Orchestrates Murine Skeletal Muscle Regeneration and Modulates Autocrine Polarization

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

92. Genetic Correction of Induced Pluripotent Stem Cells From Osteopetrotic Mice and Differentiation to the Hematopoietic Lineage as a Cell Therapy Approach

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

169. Clinically Adaptable CD19-Specific Chimeric Antigen Receptor

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

485. Protection of CD4+ T-Cells and Enhanced Immune Response Against SHIV-Challenge After Engraftment of Genetically-Modified HSCs in the Pigtailed Macaque-Aids Model

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

507. Adenoviral Vector-Mediated CFTR Gene Transfer and Anion Transport Correction of CF Pig Airways in Vivo

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

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