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Improved hFIX and hFVIII expression with ITR-modified AAV vectors in hemophilic mice

Evolution Directed Gene Engineered (EDGE) Gain-of-Function Factor VIII Variants Enhance Gene Therapy for Hemophilia A

Monthly Roundup: December 2024

Monthly Roundup: November 2024

Monthly Roundup: October 2024

Novel Approaches Under Investigation

Neutralizing Antibodies

Monthly Roundup: April 2024

A Factor VIII Variant With Elimination of the N-glycosylation Site at 2118 Reduced Anti-FVIII Immune Responses in Gene Therapy Treated Hemophilia A Mice

Stabilin-2 Promoter Modulates the Immune Response to FVIII After Lentiviral Vector Delivery in Hemophilic Mice

Delivery of CRISPR/Cas9 mRNA LNPs to Repair a Small Deletion in FVIII Gene in Hemophilia A Mice

Targeting Sinusoial Endothelium

Monthly Roundup

Interactive Webinars

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Conference Coverage

Highlights From the 17th Annual Congress of EAHAD

Highlights From the 65th ASH Annual Meeting and Exposition

Highlights From the ESGCT 30th Annual Congress

Highlights From the 31st Congress of ISTH

Highlights From the 32nd Congress of ISTH

Gene Therapy Illustrated Clinical Trials

Microlearning

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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., Roche, and Spark Therapeutics

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