“Gene Sequencing and Gene Therapy” – Matthew Skelton, PhD

Watching the videos that are posted from the conference brings back the excitement and all the feels from being at the Symposium, surrounded by a community of families, kiddos, medical professionals, researchers, and pharma companies with one thing in common: a vested interest in Cerebral Creatine Deficiency Syndromes.

During this conference session, Dr. Skelton talks about gene therapy. He explains the different avenues that can be taken with gene therapy, including modifying, replacing, and repairing genes. He talks about how gene therapy is performed (i.e. viral vector with healthy genes). Dr. Skelton also broaches the topic of if Cerebral Creatine Deficiency Syndromes are good candidates for gene therapy and what may be standing in the way for Creatine Deficiencies to benefit from gene therapy.

He concludes that there is a lot we still don’t know about gene therapy, but the hope is that we eventually could use a vector to learn more about effective treatment for creatine deficiencies.
I’d be curious to know your thoughts after watching!

**Thanks to Celeste Graham for writing this blog post.**

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