Gene therapy progress and prospects: gene therapy for the hemophilias.
Recent gene transfer trials for hemophilia A and B, bleeding disorders lacking either functional factor VIII or IX, respectively, have produced tantalizing results, suggesting that the potential to correct these bleeding disorders at a molecular level may be at hand. Genetic correction of the hemophilias represents a model system to develop a basic understanding of how gene therapy will be achieved. The goals for hemophilia gene transfer require the long-term therapeutic production of the coagulant protein without stimulating an immune response to the transgene product or the vector. Based on a scientific understanding of the molecular and cellular defects, leading to the bleeding phenotype, impressive strides have been made in the last 2 years.
Walsh
Christopher E
CE
Mt Sinai School of Medicine, One Gustave Levy Place, Rm 24-42C Annenberg Building, New York City, NY 10029, USA.
D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000235:genetics; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans; D008297:Male