Gene-Editing Therapies for the Treatment of Parkinson’s Disease

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Parkinson’s disease is associated with the accumulation of alpha-synuclein aggregates in dopaminergic neurons of the substantia nigra pars compacta, leading to cell death, and resulting loss of motor control.

Our scientists developed a method for treating Parkinson’s disease by editing the gene that encodes alphasynuclein to prevent aggregation of the encoded protein in affected cells.

Gene-Editing Therapies for the Treatment of Parkinson’s Disease is protected by two U.S. patents, as well as patents in Canada and Mexico (with additional patents pending in the U.S. and in other countries). Of note, certain granted patents include claims that are not limited by specific target sequence, type of nucleic acid, formulation, or method of transfection.

Example Applications

  • Employ multiple therapeutic strategies – monoallelic or biallelic inactivation of alpha-synuclein in vulnerable cells, altering alpha-synuclein mRNA splicing to generate non-aggregating forms by ablating splice acceptor/donor sites, etc.
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity in vivo gene editing
  • Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high efficiency in vivo delivery – proven delivery to brain in vivo

Representative Data

Figure 1. Gene editing of the SNCA gene in human iPS cells.

Representative Claim

U.S. Pat. No. 10,752,919

An in vitro method for producing a gene-edited cell comprising transfecting in vitro a cell comprising an α-synuclein (SNCA) gene with a nucleic acid encoding a gene-editing protein, wherein the gene-editing protein comprises: (a) a DNA-binding domain and (b) a nuclease domain and is capable of binding to a nucleotide sequence that encodes a protein comprising the amino acid sequence of SEQ ID NO: 51, wherein:

(a) the DNA-binding domain comprises a plurality of repeat sequences and at least one of the repeat sequences comprises the amino acid sequence: LTPvQVVAIAwxyzGHGG (SEQ ID NO: 75) and is between 36 and 39 amino acids long, wherein:

“v” is Q, D or E,

“w” is S or N,

“x” is N or H,

“y” is D, A, H, N, K, or G, and


(b) the nuclease domain comprises a catalytic domain of a nuclease, to result in a gene-edited cell.