Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice-Reference-Cited by-同舟云学术

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Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice

Published:2009-06-08 Issue: Volume: Page:
ISSN:0021-9738
Container-title:Journal of Clinical Investigation
language:en
Short-container-title:J. Clin. Invest.

Author:

Kren Betsy T.,Unger Gretchen M.,Sjeklocha Lucas,Trossen Alycia A.,Korman Vicci,Diethelm-Okita Brenda M.,Reding Mark T.,Steer Clifford J.

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Reference81 articles.

1. Progress and problems with the use of viral vectors for gene therapy

2. Gene therapy for hemophilia

3. Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment

4. LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

5. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

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1. Ultrasound-mediated gene delivery specifically targets liver sinusoidal endothelial cells for sustained FVIII expression in hemophilia A mice;Molecular Therapy;2024-04

2. Liver-Targeting Nanoplatforms for the Induction of Immune Tolerance;Nanomaterials;2023-12-26

3. Current, emerging, and potential therapies for non-alcoholic steatohepatitis;Frontiers in Pharmacology;2023-03-30

4. Defenestrated endothelium delays liver-directed gene transfer in hemophilia A mice;Blood Advances;2022-06-23

5. Three-dimensional (3D) liver cell models - a tool for bridging the gap between animal studies and clinical trials when screening liver accumulation and toxicity of nanobiomaterials;Drug Delivery and Translational Research;2022-05-04

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