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Defining therapeutic thresholds for STXBP1 gene therapy: From gene delivery to network rescue in neurodevelopmental

Yi Zheng1, Feiyang Li1

  • 1Department of Neurology, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China; Laboratory of Aging Research, School of Medicine, University of Electronic Science and Technology of China, Chengdu, China.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|June 22, 2026
PubMed
Abstract

No abstract available in PubMed .

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Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...