Compositions and methods for delivering cyclin-dependent kinase-like 5 protein

Abstract

The disclosure relates to polynucleotides, e.g., viral genomes, encoding cyclin-dependent kinase-like 5 (CDKL5), vectors, e.g., adeno-associated virus (AAV) particles, comprising said polynucleotides, compositions comprising said polynucleotides or vectors, and methods for making or delivering to a cell or subject. The polynucleotides, vectors, compositions, and methods of the present disclosure are useful for the treatment of subjects who have, have been diagnosed with having, or are at risk of having a CDKL5 deficiency disorder (CDD), developmental and epileptic encephalopathy 2, atypical Rett syndrome, and / or other CDKL5-related disorders, or at least one symptom thereof.