Hemophilia: an amazing 35-year journey from the depths of HIV to the threshold of cure.
Methods developed in the early 1970s to highly purify factor VIII (FVIII) from the plasma of large numbers of blood donors led, for the first time, to concentrates of FVIII that enabled hemophiliac to self-treat, providing independence and opening the way to safe surgery and other treatments. But, with the introduction of blood-borne viruses such as HIV-1 and hepatitis C viruses into the blood supply, these concentrates also transmitted HIV and hepatitis to a high percentage of hemophiliacs. Nevertheless, from the depths of the AIDS epidemic in hemophilia came extraordinary scientific advances that led to recombinant FVIII, the identification of HIV as the agent causing AIDS, the eventual development of effective treatments for AIDS, gene transfer approaches using lentiviruses, and treatments for hepatitis C. All of these have improved the lives of current and future hemophiliacs and have brought us to the threshold of a cure.
White
Gilbert C
GC
Blood Research Institute, BloodCenter of Wisconsin, Post Office Box 2178, Milwaukee, WI 53201-2178, USA.
D001803:Blood Transfusion / Q000266:history; D003001:Cloning, Molecular; D005169:Factor VIII / Q000235:genetics / Q000302:isolation & purification / Q000627:therapeutic use; D015316:Genetic Therapy; D015658:HIV Infections / Q000150:complications / Q000266:history* / Q000635:transmission; D015497:HIV-1 / Q000302:isolation & purification / Q000472:pathogenicity; D006467:Hemophilia A / Q000150:complications / Q000266:history* / Q000628:therapy; D016174:Hepacivirus / Q000302:isolation & purification / Q000472:pathogenicity; D049673:History, 20th Century; D049674:History, 21st Century; D006801:Humans; D015202:Protein Engineering; D011994:Recombinant Proteins / Q000235:genetics / Q000627:therapeutic use; D065227:Transfusion Reaction
D011994:Recombinant Proteins; C078147:F8 protein, human; D005169:Factor VIII