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Tea party reunion marks milestone in treatment of rare genetic disorder

After three years, Dr. Cyndi Tifft and her team of doctors reunite with Jojo, a patient with GM1 gangliosidosis who received the first gene therapy treatment for the rare disorder. Since the success of that trial, Dr. Tifft's team has treated 10 more children with GM1 gangliosidosis.

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About the National Human Genome Research Institute

At NHGRI, we are focused on advances in genomics research. Building on our leadership role in the initial sequencing of the human genome, we collaborate with the world's scientific and medical communities to enhance genomic technologies that accelerate breakthroughs and improve lives. By empowering and expanding the field of genomics, we can benefit all of humankind.

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