Genetics

3D genome architecture influences SCID-X1 gene therapy success

Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), sometimes called "bubble boy disease," are born with a defective gene that prevents them from producing immune cells. Gene therapy from St. Jude ...

Genetics

Ultrasound enables gene delivery throughout the brain

Rice University researchers tested the safety and feasibility of gene delivery to multiple brain regions using a noninvasive, ultrasound-based technique in rodents, and their findings suggest that the efficiency of gene delivery ...

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