NZCR at forefront of Gene Editing research (CRISPR/ CAS9)

New Zealand is leading the world in a new era of exciting gene research with potential to cure genetic conditions in the future.

Professor Ed Gane is running a clinical trial at NZCR to investigate a potential treatment for Transthyretin Familial Amyloidosis (FAP). FAP is characterised by abnormal build‐up of a protein called amyloid in the body’s organs and tissues. FAP is caused by a gene mutation which is hereditary, but not always passed on.

Kiwi patients are among the first in the world to benefit from a revolutionary gene‐editing treatment designed to ‘snip‐off’ a faulty heredity gene. The treatment will potentially provide a life‐long cure for the lethal disease (FAP), removing the need for future liver and heart transplant transplants with a single dose.

If it is effective, a single dose will provide lifelong cure, removing the need for future organ transplants. It could also pave the way for a new era of gene editing cures for patients with genetic conditions. The medical community is watching with anticipation the world’s first ever in‐vivo gene‐editing study.