Production of Human Factor IX in Animals by Genetically Modified Skin Fibroblasts : Potential Therapy for Hemophilia
نویسندگان
چکیده
Inherited diseases might be treated by introducing normal genes into a patient’s somatic tissues to correct the genetic defects. In the case of hemophilia resulting from a missing clotting factor. the required gene could be introduced into any cell as long as active factor reached the circulation. We previously showed that retroviral vectors can efficiently transfer genes into normal skin fibroblasts and that the infected cells can produce high levels of a therapeutic product in vitro. In the current study. we examined the ability of skin fibroblasts to secrete active clotting factor after infection with different retroviral vectors encoding human clotting factor IX. Normal human
منابع مشابه
Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B.
Inherited diseases might be treated by introducing normal genes into a patient's somatic tissues to correct the genetic defects. In the case of hemophilia resulting from a missing clotting factor, the required gene could be introduced into any cell as long as active factor reached the circulation. We previously showed that retroviral vectors can efficiently transfer genes into normal skin fibro...
متن کاملExpression of Recombinant Coagulation Factor IX in Human Amniotic Membrane-derived Mesenchymal Stem Cells: A New Strategy to Gene Therapy of Hemophilia B
Background: Hemophilia B is an X-linked hereditary disorder of blood coagulation system which is caused by factor IX (FIX) deficiency. Factor IX is a plasma glycoprotein that participates in the coagulation process leading to the generation of fibrin. Replacement of factor IX with plasma-derived or recombinant factor IX is the conventional treatment for hemophilia B to raise the factor IX le...
متن کامل[The skin as a vehicle for gene therapy: hemophilia B, an application model].
Artificial skin offers important advantages in gene therapy tor its biosafety and simple monitoring. An easy access of keratinocytes through small biopsies and their in vitro expansion enriched with epithelial stem cells, make them an ideal target for long-term therapeutic transgene expression. Corrective cutaneous gene therapy has been recently applied in clinical trials on dermatological gene...
متن کاملIn vitro Co-Culture of Human Skin Keratinocytes and Fibroblasts on a Biocompatible and Biodegradable Scaffold
Background: Extensive full-thickness burns require replacement of both epidermis and dermis. In designing skin replacements, the goal has been to re-create this model and make a product which has both essential components. Methods: In the present study, we developed procedures for establishing confluent, stratified layers of cultured human keratinocytes on the surface of modified collagen-chito...
متن کاملEvaluation of the Relationship Between Factor IX Inhibitor in Hemophilia B Patients and Different Types of Therapy in the North-eastern Part of Iran
Background: Hemophilia B is a bleeding disorder with a recessive X-linked inheritance pattern, in which the infected individuals have low levels of factor IX in their plasma. Affected individuals may have bleeding episodes after trauma or spontaneously considering the plasma level of factor IX. In order to prevent these episodes and to control bleeding, they should use coagulation factor concen...
متن کامل