gene therapy

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

Newborn screening for spinal muscular atrophy improves chances of walking and quality of life: study

18 Jan 2023

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

Testing and treating newborns for spinal muscular atrophy: saving lives and healthcare costs

03 Aug 2021

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.

Gene therapy gives families hope for babies with spinal muscular atrophy

28 Jun 2021

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

New CRISPR technology could revolutionise gene therapy, offering new hope to people with genetic diseases

02 Feb 2021

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

Light, liposomes, action: researchers show safer, more targeted way to deliver CRISPR gene therapy

12 Nov 2020

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

Boyer Lectures: gene therapy is still in its infancy but the future looks promising

15 Oct 2018

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".

New gene therapy developed with UNSW researchers targets brain disorders

09 Nov 2017

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.

A novel molecular dictator 'with a conscience' discovered

23 Jul 2007

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.