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 ...

Oncology & Cancer

An ancient anti-cancer mechanism: DISE

A new editorial paper titled "DISE, an ancient anti-cancer mechanism that senses mutational load in cancerous cells?" has been published in Oncotarget.

Genetics

Advances in gene therapy for CLN2 batten disease

A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects. The study, conducted ...

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.

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