Article
The most common disease-causing mutation of factor XIII deficiency is corrected by CRISPR/CAS9 gene editing system.
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis - 1 Apr 2022
Dorgalaleh Akbar, Kiani Jafar, Zaker Farhad, Safa Majid
Abstract excerpt
Factor XIII (FXIII) deficiency is one of the most severe congenital bleeding disorders, with an estimated incidence of one person per one million. Patients with severe FXIII deficiency present a wide range of clinical manifestations, including umbilical cord bleeding, intracranial haemorrhage and recurrent miscarriages. Due to the high rate of life-threatening bleeding, primary prophylaxis is mandatory from the...
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