New Research Suggests Gene Therapy Could Be Used To Treat Pitt-Hopkins Syndrome

Chris K

Chris K

Chris K
Staff member
Jul 3, 2023
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Postnatal gene therapy may be able to prevent or repair many of the harmful effects of Pitt-Hopkins syndrome, a rare genetic condition, according to researchers at the University of North Carolina School of Medicine. In an animal model of Pitt-Hopkins syndrome, researchers discovered that restoring lost gene activity eliminates numerous clinical indications.

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