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Publications in Molecular Medicine

136. First-In-Human Study of NS-065/NCNP-01; The Morpholino Based Antisense Oligonucleotide for Exon 53 Skipping in Duchenne Muscular Dystrophy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

Engraftment of Human Stem Cell Derived Otic Progenitors in the Damaged Cochlea

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2019English

Neurotropic Properties of AAV-PHP.B Are Shared Among Diverse Inbred Strains of Mice

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2019English

28. Mouse and Pig Photoreceptor Transduction Mediated by Triple AAV Vectors

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

506. Β-Sarcoglycan Gene Transfer Prevents Muscle Fibrosis and Inflammation in an Aged LGMD2E Mouse Model

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

219. Tracking the Fate and Activity of Individual HSC and Memory Stem T Cell Clones in GT Patients Through Insertional Tagging

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

8. An Intriguing Rep and VP-Dependent Fate of Assembly-Activating Protein During Adeno-Associated Virus Capsid Formation

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

Non-Viral, Tumor-Free Induction of Transient Cell Reprogramming in Mouse Skeletal Muscle to Enhance Tissue Regeneration

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2019English

Potentiating Oncolytic Virus-Induced Immune Mediated Tumor Cell Killing Using Histone Deacetylase Inhibition

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2019English

725. Preliminary Results of Molecular Cardiac Surgery Mediated Transfer of scAAV6-βARKct in a Reproducible Model of Ischemic Cardiomyopathy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

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