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Publications in Pharmacology

Treatment of Type 1 Myotonic Dystrophy by Engineering Site-Specific RNA Endonucleases That Target (CUG)n Repeats

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

326. Improving CRISPR-Cas9 Precision Through Directly or Indirectly Tethered DNA-Binding Domains

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

Vaccinia Virus Induces Programmed Necrosis in Ovarian Cancer Cells

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

Adoptive Cell Therapy: ACT-Up or ACT-Out?

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2019English

The Extragenic Spacer Length Between the 5′ and 3′ Ends of the Transgene Expression Cassette Affects Transgene Silencing From Plasmid-Based Vectors

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

534. Tumorigenic Effect of Transplanted Myoblasts Overexpressing BCL-2 Based on Retroviral Vector

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

110. Enhanced Intratumoral Accumulation of Nucleic Acid and Oncolytic Adenovirus by Complexation With Biocompatible and Bioreducible Polymer

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

93. Generation of Gene-Corrected, Vector- And Reprogramming Factor-Free iPS Cells From SCID-X1 Patients

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

104. Intrathoracic Administration of AAV9 Reverses Neural and Cardiorespiratory Dysfunction in Pompe Disease

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

650. The Specificity of Rhesus T Cells Can Be Redirected to CD20 Using the Sleeping Beauty Transposon/Transposase System to Express a Chimeric Antigen Receptor

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

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