I am a scientist, businessman, author, and philanthropist. For nearly two decades, I was a professor at Harvard Medical School and Harvard School of Public Health where I founded two academic research departments, the Division of Biochemical Pharmacology and the Division of Human Retrovirology.
This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged by disease, injured by trauma, or worn by time to normal function. I include a full spectrum of chemical, gene, and protein-based medicines, cell-based therapies, and biomechanical interventions that achieve that goal.
Gene therapy's history is filled with ingenuity, imagination, and exploration. The 1960s through the 1980s, in particular, were a time of significant progress, marked by breakthroughs and fundamental discoveries. During this period, there was an improved understanding of the nature of genes and their potential for curing diseases.
. She genetically altered the hemoglobin of cells taken from a sickle-cell anemia patient's bone marrow. This was done by incubating the patient's cells with DNA extracted from a donor with normal hemoglobin in tissue culture. Shortly after this, these concepts were applied practically. for a rare genetic disorder called hyperargininemia. This inherited condition is caused by a deficiency of the arginase enzyme, leading to arginine accumulation in the bloodstream. Any accumulation of arginine can cause brain damage, epilepsy, and other neurological and muscular problems. The treatment was given as a last resort to save the children's lives., and the sisters did not respond to the treatment.
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