Possible Batten disease gene therapy
Batten disease (ceroid lipofuscinosis neuronal, CLN) groups neurological disorders caused by mutations in any gene of CLN family. Gene therapies for Batten disease can follow path of restoring healthy gene expression (antisense oligonucleotides, RNA interference, gene insertion) or correcting mutated sequences (CRISPR base editors, CRISPR deletions).
News about gene therapies for Batten disease:
- 11th October 2019: Proof of concept personal RNA therapy for ultra-rare form of Batten’s disease
- 23rd October 2018: First in the world, genome-based therapy for CLN7 Batten Disease
Current experimental, genetic treatments for Batten disease:
- 2017 – ongoing: Milasen, clinical one-person trial, antisense oligonucleotide (hospital information, publication with results, foundation funding the trial)
Approved genetic treatments for Batten disease: