Genetic Therapy ; Hemophilia A ; therapy ; Humans

China ; Hemophilia A ; therapy ; Hemophilia B ; therapy ; Humans ; Quality of Life

Genetic Therapy ; Hemophilia B ; therapy ; Humans

Hemophilia is a hereditary hemorrhagic disease induced by synthesis reducing of clotting factors or functional defect because of genetic mutations, Its treatment methods include traditional replacement therapy and new types of gene therapy. Replacement therapy is to reduce the bleeding complication and prevent the loss of function through the infusion exogenous recombinant coagulation factor, and gene therapy is to import the gene that exogenous code clotting factor into the patients' body by gene transfer technology, and express the treatment level of clotting factors to achieve the purpose of the permanent cure hemophilia. The various factors which affecting effects of the hemophilia's gene therapy include carrier factors, target cell factors, the timing of treatment, immune response caused by carrier. This review summarizes briefly the research progress of the factors affecting the gene therapy for hemophilia.
Blood Coagulation Factors ; Genetic Therapy ; Hemophilia A ; Humans

Asians ; China ; Factor VIII ; Hemophilia A/therapy* ; Humans

Humans ; Hemophilia A/therapy* ; Practice Guidelines as Topic ; China

Hemophilia A(HA) is an X-linked recessive bleeding disorder caused by mutations in coagulation factor VIII. Nowadays, exogenous coagulation factor replacement therapy is the main treatment. With the continuous development of gene therapy, new research directions have been provided for the treatment of hemophilia A. CRISPR-Cas9 technology was applied to select suitable target sites, and mediate the targeted knock-in and efficient expression of exogenous B-domain-deleted FⅧ variant gene through corresponding vectors for the treatment of hemophilia A.CRISPR-Cas9 technology is an emerging gene editing tool with great efficiency, safety and effectiveness, and has been widely used in hemophilia gene therapy research. This paper reviews the vector selection, construction of therapeutic genes, gene editing technology and selection of expression target sites for hemophilia A gene therapy at this stage.
Humans ; Hemophilia A/therapy* ; CRISPR-Cas Systems ; Hemophilia B/therapy* ; Gene Editing ; Genetic Therapy ; Genetic Vectors

Anticoagulants ; pharmacokinetics ; Child ; Hemophilia A ; drug therapy ; Hemophilia B ; drug therapy ; Humans ; Precision Medicine

Asian Continental Ancestry Group ; Factor VIII ; Hemophilia A ; therapy ; Hemophilia B ; therapy ; Humans ; Practice Guidelines as Topic

Objective: To evaluate the efficacy and safety of a domestic human plasma derived coagulation Factor Ⅸ concentrate (pd-FⅨ) in patients with hemophilia B. Methods: The study was a multicenter, open-label and single-arm study. The efficacy of pd-F Ⅸ was evaluated by objective performance criteria. The doses of pd-FⅨ were calculated according to the bleeding symptom and disease severity. The infusion efficiency of pd-FⅨ and improvement of bleeding symptoms were measured at 30 minutes and (24±4) h after the first infusion, respectively. Adverse events were recorded. Viral infection and FⅨ inhibitor were detected 90 d after the first infusion. Results: All 36 subjects with hemophilia B were enrolled in the study. The median age of these patients was 31 years old and the median injection doses were 4 (1-17) times. The hemostatic effect of 27/36 (75.00%) and 9/36 (25.00%) acute bleeding events were rated as "excellent" and "better" , respectively. The recovery rate was 111.92% (65.55%-194.28%) at 30 minutes after infusion of FⅨ. There was no adverse event related to FⅨ. No reactivation of HBV, HCV or HIV and FⅨ inhibitor was detected at 90-104 d after the first FⅨ infusion. Conclusion: This domestically made human plasma derived FⅨ concentrate is safe and effective in the treatment of acute bleeding in patients with hemophilia B. Clinical trial registration: China food and Durg Administration, 2016L08027.
Adult ; China ; Factor IX ; Hemophilia A ; Hemophilia B/therapy* ; Hemorrhage ; Humans ; Plasma