Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models.

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From Virus to vector to medicine: Foreword by guest editors.Current and Future Treatments for Lysosomal Storage Disorders.CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes.Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies.

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Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models.

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2017 nî lūn-bûn

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Gene therapy with helper-depen ...... tudies in large animal models.

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Gene therapy with helper-depen ...... tudies in large animal models.

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Gene therapy with helper-depen ...... tudies in large animal models.

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Gene therapy with helper-depen ...... tudies in large animal models.

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Gene therapy with helper-depen ...... tudies in large animal models.

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Gene therapy with helper-depen ...... tudies in large animal models.

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Gene therapy with helper-depen ...... tudies in large animal models.

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Nicola Brunetti-Pierri

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Philip Ng

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The role of chromatin in adenoviral vector function

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors.

A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia.

Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A.

PEGylated helper-dependent adenoviral vectors: highly efficient vectors with an enhanced safety profile.

Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model.

Comparison of replication-competent, first generation, and helper-dependent adenoviral vaccines

Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates

Liver transplantation for glycogen storage disease types I, III, and IV

Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.

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s11262-017-1471-x

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scholarly article

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10.1007/S11262-017-1471-X

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2017-06-07T00:00:00Z

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28593513

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Adenoviridae

gene therapy

Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models.

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P2860

P2860

Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models.

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