Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment
| Autor: | David Lillicrap, Steven W. Pipe, Glenn Pierce, Thierry VandenDriessche |
|---|---|
| Přispěvatelé: | Division of Gene Therapy & Regenerative Medicine, Cell Biology and Histology |
| Rok vydání: | 2007 |
| Předmět: |
Genetic enhancement
COAGULATION Gene delivery Bioinformatics Hemophilia A Viral vector medicine Humans Vector (molecular biology) Hemophilia Blood Coagulation Factor IX Clotting factor factor IX business.industry Hematology Gene Therapy Genetic Therapy Blood Coagulation Factors Clinical trial Coagulation factor VIII Immunology business Genetic Engineering vector medicine.drug |
| Zdroj: | Journal of thrombosis and haemostasis : JTH. 5(5) |
| ISSN: | 1538-7933 |
| Popis: | The World Federation of Hemophilia estimates that of the 400,000 individuals worldwide with hemophilia, 300,000 receive either no, or very sporadic, treatment. Thus, considerable innovation will be required to provide cost-effective therapies/cures for all affected individuals. The high cost of prophylactic regimens hampers their widespread use, which further justifies the search for novel cost-effective therapies and ultimately a cure. Five gene transfer phase I clinical trials have been conducted using either direct in vivo gene delivery with viral vectors or ex vivo plasmid transfections and reimplantation of gene-engineered cells. Although there was evidence of gene transfer and therapeutic effects in some of these trials, stable expression of therapeutic factor VIII or FIX levels has not yet been obtained. Further improvements of the vectors and a better understanding of the immune consequences of gene transfer is warranted, as new trials are being initiated. Bioengineered clotting factors with increased stability and/or activity are being validated further in preclinical studies. Novel clotting factor formulations based on PEGylated liposomes with prolonged activities are being tested in the clinic, and are yielding encouraging results. |
| Databáze: | OpenAIRE |
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