Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX‐Padua-Reference-Cited by-同舟云学术

Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX‐Padua

Published:2021-03-28 Issue:5 Volume:19 Page:1212-1218
ISSN:1538-7933
Container-title:Journal of Thrombosis and Haemostasis
language:en
Short-container-title:J. Thromb. Haemost.

Author:

Robinson Mary M.¹,George Lindsey A.²,Carr Marcus E.³,Samelson‐Jones Benjamin J.²^ORCID,Arruda Valder R.²,Murphy John E.⁴,Rybin Denis⁴,Rupon Jeremy⁵,High Katherine A.²³,Tiefenbacher Stefan¹

Affiliation:

1. Colorado Coagulation Laboratory Corporation of America Holdings Englewood CO USA

2. Children’s Hospital of PhiladelphiaPerelman School of MedicineUniversity of Pennsylvania Philadelphia PA USA

3. Spark Therapeutics Inc Philadelphia PA USA

4. Pfizer Inc Cambridge MA USA

5. Pfizer Inc Collegeville PA USA

Funder

National Heart, Lung, and Blood Institute

Pfizer

Publisher

Wiley

Subject

Hematology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/jth.15281

Reference22 articles.

1. Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia

2. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

3. X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

4. Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity

5. Recombinant to modified factor VIII and factor IX - chromogenic and one-stage assays issues

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