GeneTherapy.ISTH.org - Gene Therapy Latest News

Therapeutic hFIX Activity Achieved After Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

Therapeutic hFIX Activity Achieved After Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

12 August 2019

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.

Hyperactivity of Factor IX Padua (R338L) Depends on Factor VIIIa Cofactor Activity

Hyperactivity of Factor IX Padua (R338L) Depends on Factor VIIIa Cofactor Activity

12 August 2019

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.

The Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy

The Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy

12 August 2019

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.

ISTH Announces Launch of New Global Education Initiative in Gene Therapy For Hemophilia

ISTH Announces Launch of New Global Education Initiative in Gene Therapy For Hemophilia

05 July 2019

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.

How to Discuss Gene Therapy for Haemophilia? A Patient and Physician Perspective
30 June 2019
Adenovirus-Associated Antibodies in UK Cohort of Hemophilia Patients
30 June 2019
Hemophilia Trials in the Twenty-First Century: Defining Patient Important Outcomes
30 June 2019
Phagocytosis-Shielded Lentiviral Vectors Improve Liver Gene Therapy in Nonhuman Primates
30 June 2019

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Supported by educational grants from BioMarin, Pfizer, Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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