Home / 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.
New Zealand Clinical Research (NZCR) provides state of the art research facilities and the expertise to conduct complex early phase clinical research in healthy participant and patient populations.