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Publications in Drug Discovery

150. Ex Vivo Gene Therapy of Duchenne Muscular Dystrophy Using iPSCs From Duchenne Muscular Dystrophy Patients

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

692. The Cellular Fibronectin Matrix Is a Key Factor for the Attachment of VSV-G Pseudotyped Vector Particles and Its Cell Type Specific Complexity Provides a Mechanism for Delayed Cell Entry

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2010English

370. Reconstruction of the Nigrostriatal Pathway in Parkinsonian Macaques

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

582. Expression of Therapeutic Levels of Human Clotting Factor IX in Non Human Primates From an AAV5-Based Gene Therapeutic Vector Generated Using a Fully-Scalable GMP-Compliant Production System

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

465. Quantitation of Transgene Expression With Isotope Dilution Mass Spectrometry

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2017English

223. Lentiviral Vector Mediated Gene Therapy for X-Linked Lymphoproliferative Disease Restores Humoral and Cellular Functions

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

70. Pancreatic Gene Delivery for Diabetes Therapy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

597. Therapy of Atherosclerosis in Apolipoprotein E-Deficient Mice

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

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Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

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