Engineered bacteriophage particle and method of gene therapy by using same
AssigneeAcademia Sinica
InventorsYun MOU, Chia-Yi KAO
Life of this patent
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Abstract
Provided is an engineered bacteriophage particle for gene delivery including an engineered phagemid having a restructure f1 origin of replication, an M13 phage capsid encapsulating the engineered phagemid, and a cell-targeting ligand displayed on the M13 phage capsid. Also provided is a vector system for producing the engineered bacteriophage particle and a method of gene therapy by using the engineered bacteriophage particle.
Claims28
An engineered phagemid for gene delivery, comprising:
- an f1 origin of replication including a phage packaging signal and domain A;
- and
- a transgene expression cassette configured to express a transgene in a eukaryotic cell,
- wherein the engineered phagemid has a length less than about 8,000 nucleotides and is free from encoding an M13 phage structural protein required for M13 phage assembly.
The method of claim of 25, wherein the disease susceptible to amelioration by gene therapy is a cancer or a genetic disorder.
The method of claim of 26, wherein the cancer is selected from the group consisting of skin cancer, breast cancer, head and neck cancer, lung cancer, stomach cancer, pancreatic cancer, ovarian cancer, cervical cancer, uterine cancer, kidney cancer, bladder cancer, colon cancer, prostate cancer, central nervous system cancer, ocular melanoma, neuroblastoma, multiple myeloma, and lymphoma.
The method of claim of 26, wherein the genetic disorder is selected from the group consisting of Duchenne muscular dystrophy, limb-girdle muscular dystrophy, spinal muscular atrophy, myotubular myopathy, Pompe disease, glycogen storage disease, Leber's congenital amaurosis, amyotrophic lateral sclerosis, color blindness, type 1 diabetes, and Fabry disease.
Dependencies are inferred from the claim text.