Now the field is beginning to mature and move beyond these initial tactics. The other method delivers gene treatments directly into the body, usually to easier-to-reach areas such as the eye. The more common approach draws blood from the patient and reprograms specific cells within the laboratory before reinjecting them into the person’s body. in the past five years, and numerous others, aimed at a variety of conditions, are progressing toward clinical trials.Įxisting gene therapies rely on two fundamental approaches. More than half a dozen such treatments have gained approval in the U.S. Researchers have developed different ways to correct or influence the way someone’s genes function and used those techniques to create therapies for several blood disorders, as well as degenerative eye and muscle diseases. Although the concept faced scientific and ethical uncertainty when it was floated in the 1970s, the foundation of the approach-replacing or fixing a single, disease-causing gene-has proved solid. Three decades after its first, faltering steps in humans, gene therapy is emerging as a treatment option for a small but growing number of diseases.
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