Hello to all readers out there!
This month’s research meeting was more about procedures and processes that need to be put into place.
- With the arrival of Casey’s blood samples, the development of her cell lines has started, which will be followed by CRISPR-ing and creating the CRISPER-ed cell lines. It is my understanding that this will a few months to complete.
- I have some administrative activities to complete and get the finished product back to EverlumBio
- Discussion of natural history studies explained to me that natural history studies are not patient generated, but rather come from the medical professional treating the HMERF patient. This requires HIPPA release forms from the HMERF patients, but more to come on that at a future date as I am unclear about this process. I will keep you informed as I find out more.
As a reminder to you our readers, right now the target project is to create an ASO for HMERF. Due to the length of the titin gene and the location of Casey’s and other HMERF patients occurring at exon 344, gene therapy is not a targeted treatment. The damaged gene is too far down the protein chain to allow for an effective delivery, which may create toxicity problems. Thus, the ASO is our goal. For more on ASO treatments, you can read more about them here.
Until next time, take care of yourselves!
Gratefully,
Chris Duane, Casey’s mom… ????
