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Gene therapy

Towards a Clinically Relevant Lentiviral Transduction Protocol for Primary Human CD34⁺ Hematopoietic Stem/Progenitor Cells

Michelle Millington, Allison Arndt, [ ... ], Sylvie Shen

An Efficient Large-Scale Retroviral Transduction Method Involving Preloading the Vector into a RetroNectin-Coated Bag with Low-Temperature Shaking

Katsuyuki Dodo, Hideto Chono, [ ... ], Junichi Mineno

Chitosan-Graft-Polyethylenimine/DNA Nanoparticles as Novel Non-Viral Gene Delivery Vectors Targeting Osteoarthritis

Huading Lu, Yuhu Dai, Lulu Lv, Huiqing Zhao

Delivery of Full-Length Factor VIII Using a piggyBac Transposon Vector to Correct a Mouse Model of Hemophilia A

Hideto Matsui, Naoko Fujimoto, [ ... ], Akitsu Hotta

Bicistronic Lentiviruses Containing a Viral 2A Cleavage Sequence Reliably Co-Express Two Proteins and Restore Vision to an Animal Model of LCA1

Jonathan D. Verrier, Irina Madorsky, [ ... ], Susan L. Semple-Rowland

AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

Vidyullatha Vasireddy, Jason A. Mills, [ ... ], Jean Bennett

Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis

Sadik H. Kassim, Hui Li, [ ... ], Daniel J. Rader

Destabilizing Domains Mediate Reversible Transgene Expression in the Brain

Khalid Tai, Luis Quintino, [ ... ], Cecilia Lundberg

MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy

Mojgan Rastegar, Akitsu Hotta, [ ... ], James Ellis

Hair Cell Regeneration after ATOH1 Gene Therapy in the Cochlea of Profoundly Deaf Adult Guinea Pigs

Patrick J. Atkinson, Andrew K. Wise, [ ... ], Rachael T. Richardson

Correction of Diabetic Hyperglycemia and Amelioration of Metabolic Anomalies by Minicircle DNA Mediated Glucose-Dependent Hepatic Insulin Production

Tausif Alam, Philip Wai, [ ... ], Hans Sollinger

In Vivo Electroporation Mediated Gene Delivery to the Beating Heart

Erick L. Ayuni, Amiq Gazdhar, [ ... ], Hendrik T. Tevaearai

High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells and Erythroid Lineage-Restricted Expression by Optimized AAV6 Serotype Vectors In Vitro and in a Murine Xenograft Model In Vivo

Liujiang Song, Xiaomiao Li, [ ... ], Arun Srivastava

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