Finding cures for children's genetic diseases

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Henry and his sister face a serious genetic disesase that affects every aspect of their lives. 

Curing genetic disease is hard and complex work, but is what Dr Lisowski describes as a “deep” but solvable problem.

By donating today, you can help change the future for kids like Henry.

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1 in 20 kids is born with a birth defect or genetic disease, and with over 6,000 different types of genetic diseases, it may seem like a problem that’s too hard to deal with.

 

Each year many thousands of Australian children are affected, children like Henry and Rosalie who bear the burden of a barely-pronounceable genetic disease so serious, it affects every aspect of their lives.

A single change in a DNA can have devastating, life-changing consequences for children and families.

Today, there are no established cures for genetic diseases. Not yet.

 

Dr Leszek Lisowski and his team here at the Institute are building the tools to achieve these cures. And they are excited about the future.

 

 Dr. Leszek Lisowski, one of the brilliant scientists at the Children’s Medical Research Institute devotes himself tirelessly to help find cures for genetic diseases affecting those 1 in 20 children.


It is hard and complex work, but curing genetic disease is what Dr Lisowski describes as a “deep” but solvable problem.

Dr Lisowski is developing “gene therapy” techniques, the most promising approach toward curing genetic diseases.

Leszek’s team develops tools called ‘vectors’ – miniature biological machines that can deliver a healthy (typo-free) copy of a gene into diseased cells or locate errors in DNA and correct the typo.

Importantly, by targeting the underlying cause of a genetic disease—and correcting the error within the gene—gene therapy can lead to cures.

Not just a treatment, but a lasting cure.

Leszek’s goal now is to create Australia’s first dedicated laboratory to produce the high-quality, clinical-grade vectors he needs to help cure children affected by genetic diseases.

 

Dr Lisowski has committed his entire career to vector-based gene therapy. He’s an expert at making vectors. But he cannot do it alone.

We are seeking to raise $580,000 by the by the end of financial year (30 June) to ensure this work continues. Be a supporter of this important work of Dr Leszek Lisowski and his team.

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Like most people, Jess was excited to have her first child. Like most people, she assumed she would have a healthy baby. When she realised Henry was born with LCHADD, that all changed.

Read Jess's Story