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 ...

Alzheimer's disease & dementia

Using focused ultrasound to treat Alzheimer's and Parkinson's

The Ultrasound and Elasticity Imaging Laboratory, led by Elisa Konofagou, Robert and Margaret Hariri Professor of Biomedical Engineering, develops novel, ultrasound-based techniques for both imaging and therapeutic applications.

Diseases, Conditions, Syndromes

New study shows promising evidence for sickle cell gene therapy

New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).

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