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New Gene Editing Technology (CRISPR)

Who: Children’s Hospital at Westmead and Murdoch Children’s Research Institute

Where: Sydney

About:

In collaboration with the Children’s Hospital at Westmead and the Murdoch Children’s Research Institute (MCRI), a research project has been developed that uses new gene editing technology (CRISPR) in the hope of a new treatment approach for NF1.   

An application for funding was successfully made to the US CTF to support this. The Centre for Translational Research (funded by the CTF Australia) will provide the clinical support, with laboratory support to come from the MCRI in Melbourne.


Funded projects

Tint: Mek Inhibitor trial
New Gene Editing Technology (CRISPR)
Neurofibromatosis Type 1 Severity Scale
Children with NF1 & Auditory Processing Deficits + Speech Perception Problems
Predicators of Autism in NF1: Development in Adolescence (PANDA)
Documenting and characterising the various MRI brain changes
Cell-free Bio bank