gene therapy

A 3D-rendering showing a cross-section of a liposome

Biomedical researchers have come up with a novel way to use a beam of light to deliver CRISPR gene therapy molecules targeting illnesses.

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Once genetic lesions for diseases such as cystic fibrosis and haemophilia were identified, the idea of replacing or correcting defective genes grew into what we now call "gene therapy".

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UNSW researchers have tailored gene therapy for a rare genetic disorder called Canavan disease, and say it could eventually be adapted to treat other inherited disorders affecting the brain.

Molecular structure inside

UNSW researchers have uncovered an important naturally occurring mechanism in the body where "bad" cells that cause blockages in our blood vessels are kept under strict growth control, while "good" cells that keep our blood vessels free of clots and growths are left unaffected.