This approach tests various pharmacological molecules that are already being used for other types of therapy to see if they can be effective in treating CDKL5. Currently, there are some trials being done to test toxicity and neurological benefits. Theses results bring hope that these medications may improve cognitive abilities in children with CDKL5 syndrome.
Protein substitution therapy
Protein substitution therapy aims to substitute the missing or malfunctioning protein CDKL5, with a lab created version Tatk-CDKL5. This study seems to be the most promising one. The lab created protein seems to be able to adequately substitute for the original one, thus allowing for the neuronal development to resume. The major challenges to this method are the production costs. We are currently focused on finding a source that can scale up production without compromising stability or purity.
Gene therapy consists in transferring genetic material to prevent or cure a disease. In the case of genetic disorders, the therapy consists in substituting the mutated or malfunctioning gene with a functioning one. Genetic therapy is highly innovative and thanks to recent scientific progress it is now a possible treatment method even for genetically based neurological disorders.
A genetically engineered viral vector is the most effective method to transfer DNA inside the sick cell.