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122. Seamless Correction by Donor DNA of a Class I CFTR Mutation Facilitated by a Double Nicking CRISPR/Cas9 in CF-iPSCs

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

857. Donor Dependency of Integrin-And Chemokine Mediated Adhesion Activation in Human Mesenchymal Stromal Cells (MSCs)

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2011English

203. Phase I Study of the “Triad” Autologous (FANG™) Vaccine, Incorporating Bifunctional shRNAfurin and GMCSF Transgene Expression, in Advanced Ewing's Sarcoma: Preliminary Data in Pediatrics Patients

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

Research Highlights

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2012English

69. Combination Therapy of Reovirus and PD-1 Blockade Effectively Establishes Tumor Control via Innate and Adaptive Immune Responses

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

404. Skeletal Muscle Engraftment of Mesenchymal Stromal Cells Is Augmented by IL-10

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

766. Genetic Correction of Ifnγr1 Deficiency in Hematopoietic Cells Repairs the Cellular Phenotype of Mendelian Susceptibility to Mycobacterial Disease

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2016English

37. Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Adrenoleukodystrophy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

311. Expansion of Gene-Modified T Cells Following Direct Intravenous Injection of Therapeutic Foamy Virus Vector in a Canine Model of Severe Combined Immunodeficiency

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

395. Tumor Recurrences Share Immunogenic Antigens Across Both Tumor Types and Primary Treatments Which Can Be Therapeutically Targeted With VSV-cDNA Libraries

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2013English

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