In a milestone year, gene therapy finds a place in medicine

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In a milestone year, gene therapy finds a place in medicine

Brian Madeux sits with his girlfriend Marcie Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Children’s Hospital in Oakland, Calif. Madeux, who has Hunter syndrome, received the treatment on Nov. 13. Gene therapy aims to treat the root cause of a problem by deleting, adding or altering DNA, rather than just treating symptoms that result from the genetic flaw.
Dr. Albert Maguire, right, checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelphia. Misa was 4-years-old when he received his gene therapy treatment. On Dec. 19, the Food and Drug Administration approved the therapy which improves the vision of patients with a rare form of inherited blindness, another major advance for the burgeoning field of genetic medicine.

In a milestone year, gene therapy finds a place in medicine

Brian Madeux sits with his girlfriend Marcie Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Children’s Hospital in Oakland, Calif. Madeux, who has Hunter syndrome, received the treatment on Nov. 13. Gene therapy aims to treat the root cause of a problem by deleting, adding or altering DNA, rather than just treating symptoms that result from the genetic flaw.
Dr. Albert Maguire, right, checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelphia. Misa was 4-years-old when he received his gene therapy treatment. On Dec. 19, the Food and Drug Administration approved the therapy which improves the vision of patients with a rare form of inherited blindness, another major advance for the burgeoning field of genetic medicine.