We just received amazing news from Cure AP-4 about the status of the proof of concept for SPG52 gene therapy!
Dr. Mimoun Azzouz, Cure AP-4’s Principal Investigator working on AP4B1 gene therapy research began working on a gene therapy proof-of-concept for SPG52 a few months ago using internal University of Sheffield funds available to his lab, since Dr. Azzouz has learned a great deal about AP-4-HSP through his efforts to develop a gene therapy treatment for SPG47 over the past 2+ years.
As of now, the AP4S1 viral constructs are nearly complete and AP4S1 knock-out mouse model has been created through a group in the UK who has aided in the funding of the models. Once the viral constructs are complete, he can begin toxicity studies on the wild type mice (those are mice with two working AP4S1 genes as you would likely find in the wild). He is on track to begin injecting the AP4S1 knock-out mouse colony by the end of March 2020.
This is the point where more funding will be essential, as it is becomes costly to maintain the mice and the research that goes along with them.
We’re so excited by this news and kicking our fundraising into high gear to help keep this research moving!
Please consider a tax deductible donation to Cure SPG52 today. Thank you!
http://cureap4.org/donations.php#spg52