Gene Editing Technology and Therapeutics Laboratory

Re-writing the future of genetic disease

Many children are born with genetic diseases, and for many of these conditions, there is still no cure. This is devastating for both the child and their families. We believe that every person born with a genetic disease deserves the chance for a cure. That belief drives everything we do.

Led by NHMRC Investigator Fellow Dr Fatwa Adikusuma, the Gene Editing Technology and Therapeutics Laboratory is dedicated to advancing CRISPR gene editing technology and developing the next generation of gene editing therapies for people living with genetic diseases.

CRISPR gene editing has the power to correct the root cause of genetic disease at the DNA level. Unlocking this potential for patients requires precise and efficient correction of disease-causing mutations, as well as safe and effective delivery of the editing machinery to the right tissues in the body. These are the challenges our research program is built to solve.

We work on two fronts: developing novel gene editing technologies that push the boundaries of what CRISPR can do; and translating those advances into effective therapies. We currently focus on three conditions - Duchenne muscular dystrophy (DMD), autosomal dominant retinitis pigmentosa (adRP), and cystic fibrosis (CF) - each pursued in close partnership with collaborators and clinicians. Further, we are actively expanding into new disease areas through our own research and further collaborations.

We host the Australian Gene Editing Network (AGENt) - a national community connecting researchers across the gene editing field. We welcome prospective students, researchers, and collaborators who share our passion for bold, meaningful science.

Gene editing

CRISPR technologies

Genome engineering

Genetic disease therapeutics

Gene therapy

Cell and animal disease models

Explore the Gene Editing Technology and Therapeutics Laboratory

Research impact Research impact
Our people Our people
Current projects Current projects

Advancing CRISPR science transforms lives

Gene editing has the power to cure genetic diseases by correcting faults in DNA. CRISPR, the technology that enables precise gene editing, holds enormous promise but still requires significant improvements to achieve safe, efficient, and reliable therapeutic outcomes. Our laboratory is dedicated to advancing CRISPR technology to make gene editing highly effective for the treatment of genetic diseases.

Building better gene editing tools

We have developed a series of innovations that researchers around the world now use in their own work. Our tools and techniques have been adopted globally - with over 580 of our gene editing technology materials distributed to research laboratories across the world. Our discoveries have also helped the scientific community better understand the risks of gene editing, making the technology more reliable.

Developing therapies for patients

We are developing CRISPR therapies for three serious genetic diseases:

Duchenne Muscular Dystrophy (DMD)

A devastating muscle-wasting disease affecting young boys. We have developed a promising CRISPR strategy that has shown encouraging results in patient cells and animal models.

Retinitis Pigmentosa (adRP)

An inherited disease causing progressive vision loss. We have developed and patented a novel CRISPR approach that both stops the harmful protein causing the disease and restores normal function.

Cystic Fibrosis (CF)

The most common life-limiting genetic disease in Australia. We are developing a single CRISPR therapy strategy that could treat all CF patients, regardless of their specific mutation.

CRISPR gene editing is a powerful tool, but it is not perfect. Edits are not always efficient, some genomic sites are difficult to access, the editing machinery can be too large to deliver, and off-target edits can occur. Our laboratory develops novel strategies and technologies to overcome these challenges - making gene editing more precise, efficient, accessible, and safe. This foundational work underpins everything we do and benefits the broader research community worldwide.