Batten disease – Treatment with Gene Therapy


Batten disease (ceroid lipofuscinosis neuronal, CLN): a group of 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:


Current approved genetic treatments for Batten disease: