Methods to regulate polarization and enhance function of cells
First Claim
1. A method for at least one of editing or regulating a gene in a target excitable cell, the method comprisingadministering to a patient in need thereof a plurality of nanoparticles coated with a biocompatible molecule for cell uptake, the nanoparticles conjugated with at least one opsin family gene, a second unmutated gene other than the opsin family gene which ameliorates a genetic or acquired degenerative disease or condition, an antibody that targets the nanoparticles to the target excitable cell, and a CRISPR/cas9 complex to form a complex of coated nanoparticle-gene-CRISPR/cas9;
- delivering the nanoparticle-gene-CRISPR/cas9 complex to the target cell using the nanoparticle of the complex as a carrier without using a viral vector, and stimulating the complex and the target excitable cell with one or more energy sources under conditions sufficient to introduce the at least one opsin-family gene, the second gene, and the CRISPR/cas9 complex into the target excitable cell;
using the CRISPR/cas9 complex to excise from the DNA of the target excitable cell a DNA segment bearing a mutation inducing the genetic or acquired degenerative disorder or condition; and
replacing the excised mutated DNA segment with the second unmutated gene to treat the patient;
wherein stimulating the complex and the target excitable cell with one or more energy sources includes stimulating the nanoparticle of the complex and the target excitable cell with light.
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Abstract
Methods and compositions to controllably regulate cells at a target site. A quantum dot-targeting agent complex is administered to a patient in need of therapy, and the complex is stimulated using an implanted fiber optic system. In embodiments, the system includes an electrical sensor that detects and monitors electrical activity of the stimulated controllably regulated cells, and relays this information to a controller that can regulate further stimulation.
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Citations
17 Claims
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1. A method for at least one of editing or regulating a gene in a target excitable cell, the method comprising
administering to a patient in need thereof a plurality of nanoparticles coated with a biocompatible molecule for cell uptake, the nanoparticles conjugated with at least one opsin family gene, a second unmutated gene other than the opsin family gene which ameliorates a genetic or acquired degenerative disease or condition, an antibody that targets the nanoparticles to the target excitable cell, and a CRISPR/cas9 complex to form a complex of coated nanoparticle-gene-CRISPR/cas9; -
delivering the nanoparticle-gene-CRISPR/cas9 complex to the target cell using the nanoparticle of the complex as a carrier without using a viral vector, and stimulating the complex and the target excitable cell with one or more energy sources under conditions sufficient to introduce the at least one opsin-family gene, the second gene, and the CRISPR/cas9 complex into the target excitable cell; using the CRISPR/cas9 complex to excise from the DNA of the target excitable cell a DNA segment bearing a mutation inducing the genetic or acquired degenerative disorder or condition; and replacing the excised mutated DNA segment with the second unmutated gene to treat the patient; wherein stimulating the complex and the target excitable cell with one or more energy sources includes stimulating the nanoparticle of the complex and the target excitable cell with light. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16)
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17. A method for at least one of editing or regulating a gene in a target excitable cell, the method comprising administering to a patient in need thereof a plurality of nanoparticles coated with a biocompatible molecule for cell uptake, the nanoparticles conjugated with at least one opsin-family gene, a second unmutated gene other than the opsin family gene which ameliorates a genetic or acquired degenerative or condition, an antibody that targets the nanoparticles to the target excitable cell, and a CRISPR/cas9 complex to form a complex of coated nanoparticle-gene-CRISPR/cas9, the nanoparticles including piezoelectric nanoparticles;
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delivering the nanoparticle-gene-CRISPR/cas9 complex to the target excitable cell using the nanoparticle of the complex as a carrier without using a viral vector and stimulating the complex and the target excitable cell with an energy source under conditions sufficient to introduce the at least one opsin-family gene, the second unmutated gene, and the CRISPR/cas9 complex into the target excitable cell; and using the CRISPR/cas9 complex to excise a DNA segment bearing a mutation inducing the genetic or acquired degenerative or condition from the target excitable cell; and replacing the excised mutated DNA segment with the second unmutated gene to treat the patient; wherein stimulating the complex and the target excitable cell further comprises remotely controlling the piezoelectric nanoparticles with an ultrasound probe to wirelessly stimulate the piezoelectric nanoparticles and control a diode laser surgically implanted in the patient to produce laser light for stimulating the target excitable cell expressing the at least one opsin-family gene, the piezoelectric nanoparticles acting as an “
on”
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off”
switch of a battery supplying electricity to the diode laser, and wherein a ribbon of piezoelectric nanoparticles are attached to the battery of the diode laser that, upon ultrasound stimulation, creates a pulsating electrical current in the piezoelectric nanoparticles attached to the battery of the diode laser, thus controlling activation of the diode laser light production.
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Specification