Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy
نویسندگان
چکیده
منابع مشابه
Coagulation Factor IX for Hemophilia B Therapy
Factor IX is a zymogen enzyme of the blood coagulation cascade. Inherited absence or deficit of the IX functional factor causes bleeding disorder hemophilia B, which requires constant protein replacement therapy. Reviewed herein are the current state in the manufacturing of FIX, improved variants of the recombinant protein for therapy, transgenic organisms for obtaining FIX, and the advances in...
متن کاملBioengineering coagulation factors for improved hemophilia treatments☆
The implementation of prophylaxis with regular factor VIII (FVIII) replacement for patients with hemophilia radically changed the clinical presentation of this condition, from a disease characterized by progressive disabling musculoskeletal complications, to one compatible with an active and virtually normal lifestyle. In Brazil, although most adults with severe hemophilia still suffer the impa...
متن کاملRecent Advances in Hemophilia Gene Therapy
Heterogeneous loss of function mutations at F8 and F9 genes causes X-linked recessive bleeding disorders, hemophilia A (HA) and hemophilia B (HB), respectively. HA is clinically indistinguishable from HB and accounts for more than 80% of hemophilia cases; the former affects 1/5000 and the latter 1/25000 male births worldwide. In Iran, it is estimated that around 4300 HA and 900 HB patients are ...
متن کاملGene therapy for hemophilia.
Hemophilia is an X-linked inherited bleeding disorder consisting of two classifications, hemophilia A and hemophilia B, depending on the underlying mutation. Although the disease is currently treatable with intravenous delivery of replacement recombinant clotting factor, this approach represents a significant cost both monetarily and in terms of quality of life. Gene therapy is an attractive al...
متن کاملGene therapy for hemophilia.
Hemophilia A and B are X-chromosome linked recessive bleeding disorders that result from a deficiency in factor VIII (FVIII) and factor IX (FIX) respectively. Though factor substitution therapy has greatly improved the lives of hemophiliac patients, there are still limitations to the current treatment that have triggered interest in alternative treatments by gene therapy. Significant progress h...
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ژورنال
عنوان ژورنال: Molecular Therapy - Methods & Clinical Development
سال: 2019
ISSN: 2329-0501
DOI: 10.1016/j.omtm.2018.12.007