Treatment for nucleotide repeat expansion disease
AssigneeThe Johns Hopkins University
InventorsJiou Wang, Honghe Liu
Life of this patent
Estimating the term…
Abstract
Provided herein is technology relating to nucleotide repeat elements present in the genomes of eukaryotes and particularly, but not exclusively, to technologies for reducing the levels of disease-causing products expressed from expansions of nucleotide repeats.
Claims90
A ribonucleoprotein (RNP) comprising a Cas13 protein and a gRNA, wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
A nucleic acid comprising a first nucleotide sequence encoding a Cas13 protein and a second nucleotide sequence encoding a gRNA, wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
A system comprising a Cas13 protein and a gRNA, wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
A system comprising a nucleic acid encoding a Cas13 protein and a gRNA, wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
A system comprising a Cas13 protein and a nucleic acid encoding a gRNA, wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
A system comprising a nucleic acid encoding a Cas13 protein and a nucleic acid encoding a gRNA, wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
A method of treating a subject having a neurological disease, said method comprising administering a ribonucleoprotein (RNP) to said subject, wherein said RNP comprises a Cas13 protein and a gRNA and wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
A method of treating a subject having a neurological disease, said method comprising administering a nucleic acid to said subject, wherein said nucleic acid comprises a first nucleotide sequence encoding a Cas13 protein and a second nucleotide sequence encoding a gRNA and wherein said gRNA comprises a spacer sequence comprising one or more CCGGCC sequences.
The method of claim 78, wherein said Cas13 protein is Cas13d.
Dependencies are inferred from the claim text.