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Publications in Molecular Medicine

From Selection Hits to Clinical Leads: Progress in Aptamer Discovery

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2016English

T Cell-Derived iPSCs as a Source of Regenerated T Cells: High Frequency Production of iPSC Clones Capable of Generating Potent Cytotoxic T Cells

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2020English

Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2018English

Pharmacology of Recombinant Adeno-Associated Virus Production

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2018English

Bone-Targeted Alkaline Phosphatase Treatment of Mandibular Bone and Teeth in Lethal Hypophosphatasia via an scAAV8 Vector

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2018English

Development of a Novel Recombinant Adeno-Associated Virus Production System Using Human Bocavirus 1 Helper Genes

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2018English

Platelet-Targeted Gene Therapy for Hemophilia

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2018English

An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8 + T Cells

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2017English

Serum-Free Erythroid Differentiation for Efficient Genetic Modification and High-Level Adult Hemoglobin Production From Primary Human Progenitors

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2018English

Assessment of Toxicity and Biodistribution of Recombinant AAV8 Vector–mediated Immunomodulatory Gene Therapy in Mice With Pompe Disease

Molecular Therapy - Methods and Clinical Development
Molecular MedicineGeneticsMolecular Biology
2019English

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