Fundamental of Gene Therapies

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

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

AAV Integration Summary and Implications

Breaking Transgene Tolerance via Immunosuppression

Lentiviral Gene Therapy to the Liver for Hemophilia and Beyond

Overcoming anti-AAV pre-existing immunity to achieve safe and efficient gene transfer in clinical settings

Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing

52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: Data From the Phase 3 HOPE-B Gene Therapy Trial

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model

Monthly Roundup

Interactive Webinars

Podcasts

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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