Adeno-Associated Virus Delivery of CLN8 PolynucleotideAbout 3 of every 100,000 births are affected by Batten disease, a disorder that prevents the body from removing cellular waste like lipids and proteins. The build up of cellular waste throughout the body can cause seizures, vision loss, delays in thinking, abnormal movements and death. Researchers at Nationwide Children’s Hospital created a new intrathecal gene therapy using Adeno-Associated viral vectors for Batten Disease caused by mutations in the CLN8 gene. They generated an Adeno-Associated viral vector serotype 9 (AAV9) that contains the human CLN8 cDNA driven by a promoter. Additionally, they designed a new concept of intrathecal dosing which calculates the dosage by the size and growth rate of the nervous system and cerebrospinal fluid. The intrathecal gene therapy method can be used for Batten disease caused by mutations in the CLN8 gene along with other gene therapy applications for other neurological and neurodegenerative diseases. |
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