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:

Current experimental, genetic treatments for Batten disease:

Approved genetic treatments for Batten disease:

  • none