gene therapy

newborn baby in hospital bed lying on their back with hospital bracelets on feet

Testing in the newborn period is more effective than conventional clinical diagnosis pathways for improving health outcomes in infants with the rare genetic disease.

Close-up of newborn baby's feet poking out of blanket

Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.

New born baby

A trial has revealed that babies with SMA treated with gene therapy are meeting the normal developmental milestones of any baby.

Gene graphic with human figures

Using 'base editing', researchers have cured progeria in mice. This genetic syndrome causes premature ageing in humans – those with the disease usually don't live past the age of 13.

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.