Gene Therapy without using viruses to get genes to cells

Gene Therapy goal: deliver desired genes to cells to either cure disease or improve body functions such as stronger muscles. Gene therapy has the potential to enhance human performance and cure diseases.

Problem: Viruses used for gene delivery occasionally cause severe immune responses.

The novel lipid molecule created at UC Santa Barbara has a tree-shaped, nanoscale headgroup and displays unexpectedly superior DNA-delivery properties.

Background: Lipids are molecules with two parts, a water-liking “headgroup” and oily tails that assemble together to avoid water. Lipids, along with carbohydrates and proteins, constitute the main structural material of living cells.


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