An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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wetenschappelijk artikel

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наукова стаття, опублікована у вересні 2018

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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P1476

An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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P2093

Chengwen Li

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Clinton D Lothrop

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Elizabeth P Merricks

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Glenn P Niemeyer

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Junjiang Sun

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Lauren Wimsey

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Paul E Monahan

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R Jude Samulski

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Timothy C Nichols

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Wenwei Shao

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P2860

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Early clinical data raise the bar for hemophilia gene therapies.

Hepatic gene transfer as a means of tolerance induction to transgene products

Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application.

Inhibitors - cellular aspects and novel approaches for tolerance.

Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.

P304

257-267

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P31

scholarly article

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10.1016/J.OMTM.2018.07.011

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P407

English

P478

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P577

2018-09-21T00:00:00Z

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P698

30140713

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P932

6104583

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