Abstract
The present disclosure provides methods of using a prime editor in combination with prime editing guide RNAs (pegRNAs) and, optionally, nicking gRNAs to carry out prime editing to directly correct mutations (i.e., c.1412delA) in the CDKL5 gene that cause CDKL5 deficiency disorder. The present disclosure also provides compositions, complexes, and systems comprising the pegRNAs, nicking gRNAs, and/or prime editors disclosed herein, as well as polynucleotides and vectors encoding the same, and cells, kits, and pharmaceutical compositions comprising the same.
Full Text
What is claimed is:
The present disclosure provides methods of using a prime editor in combination with prime editing guide RNAs (pegRNAs) and, optionally, nicking gRNAs to carry out prime editing to directly correct mutations (i.e., c.1412delA) in the CDKL5 gene that cause CDKL5 deficiency disorder. The present disclosure also provides compositions, complexes, and systems comprising the pegRNAs, nicking gRNAs, and/or prime editors disclosed herein, as well as polynucleotides and vectors encoding the same, and cells, kits, and pharmaceutical compositions comprising the same.
Timeline
Filed
05/01/2026Published
08/20/2026Granted
Not AvailableIPC Codes(9)
C12N 15/113:Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/7105:Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 48/00:Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy