Patent ID: 11932668
Assignee: CALIFORNIA INSTITUTE OF TECHNOLOGY
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 1:
2. The method of claim 1, wherein the nucleic acid sequence to be delivered comprises one or more of:
a) a DNA sequence that encodes a trophic factor, a growth factor, or other soluble factors capable of being released from the transduced cells and affect the survival or function of that cell and/or surrounding cells;
b) a cDNA that restores protein function to humans or animals harboring a genetic mutation(s) in that gene;
c) a cDNA that encodes a protein that can be used to control or alter the activity or state of a cell;
d) a cDNA that encodes a protein or a nucleic acid used for assessing the state of a cell;
e) a cDNA and/or associated guide RNA for performing genomic engineering;
f) a sequence for genome editing via homologous recombination;
g) a DNA sequence encoding a therapeutic RNA;
h) a shRNA or an artificial miRNA delivery system; and
i) a DNA sequence that influences the splicing of an endogenous gene.