piggyBac-mediated phenotypic correction of factor VIII deficiency
نویسندگان
چکیده
منابع مشابه
piggyBac-mediated phenotypic correction of factor VIII deficiency
Hemophilia A, caused by a deficiency in factor VIII (FVIII), is the most severe inherited bleeding disorder. Hemophilia A is an attractive gene therapy candidate because even small increases in FVIII levels will positively alter the phenotype. While several vectors are under investigation, gene addition from an integrated transgene offers the possibility of long term expression. We engineered t...
متن کاملCombined Factor V and VIII Deficiency
This review summarizes current data on the pathomechanisms and new genetic findings of combined factor V and VIII deficiency (CF5F8D). Congenital haemorrhagic disorders characterized by deficiency of two clotting factors comprise an interesting group. Among dual coagulation disorders, CF5F8D is the most common type. For the first time combined factor V and VIII deficiency (F5F8D) was reported b...
متن کاملFACTOR V AND VIII INHIBITOR IN PATIENTS WITH COMBINED FACTOR V AND VIII DEFICIENCY
Patients with coagulation factor(s) deficiency who use coagulation therapy are susceptible to forming inhibitors against coagulation factor(s). In this survey we detected factor V and VIII inhibitor in ten patients with combined deficiency of factors V and VIII from north east of Iran (Khorassan province). It was revealed in our survey that eight patients had both factor V and factor VIII i...
متن کاملHaemophilia A (Factor VIII Deficiency)
This is a bleeding disorder caused by deficiency of clotting factor VIII. The vast majority of cases are inherited but acquired forms do exist, largely in older patients, due to autoantibodies directed against factor VIII or haematological malignancy. [2] Severity of disease depends upon levels of remaining factor activity, with normal range expressed as 50-200% (refer to local laboratory for r...
متن کاملHaemophilia A (Factor VIII Deficiency)
This is a bleeding disorder caused by deficiency of clotting factor VIII. The vast majority of cases are inherited but acquired forms do exist, largely in older patients, due to autoantibodies directed against factor VIII or haematological malignancy. [2] Severity of disease depends upon levels of remaining factor activity, with normal range expressed as 50-200% (refer to local laboratory for r...
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ژورنال
عنوان ژورنال: Molecular Therapy - Methods & Clinical Development
سال: 2014
ISSN: 2329-0501
DOI: 10.1038/mtm.2014.42