IP Library › Granted Patent US 12697360
Granted Patent B2
US 12697360 · App. 18/097,096 · Granted Aug 4, 2026

Viral vectors for treating Parkinson's disease

Inventors: Tyler Lahusen (Rockville, MD); Charles David Pauza (Rockville, MD)
Assignee: American Gene Technologies International Inc.
A61K35/76A61K31/7105C12N9/1077C12N15/1137C12N15/86A61K45/06C12N2310/14C12N2310/531C12N2320/32C12N2330/51C12N2740/16043C12Y204/0203
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Quick Facts
Patent No.
US 12697360
App. No.
18/097,096
Granted
Aug 4, 2026
Kind
B2
Abstract

A lentiviral vector system for expressing a lentiviral particle is disclosed. The lentiviral vector system includes a therapeutic vector, an envelope plasmid, and at least one helper plasmid. The lentiviral vector system can produce a lentiviral particle for inhibiting PARP expression in neuron cells of a subject afflicted with Parkinson's disease.

Claims (19)

1 . A method of treating a subject suffering from Parkinson's disease, comprising administering to the subject a lentiviral particle that comprises:

an envelope protein capable of infecting a cell in the subject; and

a shRNA having at least 80% sequence identity with SEQ ID NO.: 6 or 7.

2 . The method of claim 1 , wherein the cell comprises a neuron.

3 . The method of claim 2 , wherein the neuron comprises a tyrosine hydroxylase (TH+) neuron.

4 . The method of claim 1 , further comprising a second therapeutic regimen.

5 . The method of claim 1 , further comprising a second therapeutic regimen comprising ablative surgical intervention, neural stimulation, administration of L-DOPA or administration of a dopamine agonist.

6 . The method of claim 1 , wherein the envelope protein comprises a neuron-specific sequence.

7 . The method of claim 6 , wherein the neuron-specific sequence targets the shRNA to a neuron.

8 . The method of claim 6 , wherein the neuron-specific sequence encodes at least one of vesicular stomatitis virus G glycoprotein (VSV-G), fusion glycoprotein type C (FUG-C), and gp64.

9 . A method of decreasing poly(ADP-ribose) polymerase-1 (PARP-1) expression, comprising administering to a subject a lentiviral particle comprising:

An envelope protein capable of infecting a cell in the subject; and

A shRNA having at least 80% sequence identity with SEQ ID NO: 6 or 7.

10 . The method of claim 9 , wherein the cell comprises a neuron.

11 . The method of claim 10 , wherein the cell comprises a tyrosine hydroxylase (TH+) neuron.

12 . The method of claim 9 , further comprising a second therapeutic regimen.

13 . The method of claim 9 , further comprising a second therapeutic regimen comprising ablative surgical intervention, neural stimulation, administration of L-DOPA or administration of a dopamine agonist.

14 . The method of claim 9 , wherein the envelope protein comprises a neuron-specific sequence.

15 . The method of claim 14 , wherein the neuron-specific sequence encodes at least one of vesicular stomatitis virus G glycoprotein (VSV-G), fusion glycoprotein type C (FUG-C), and gp64.