| Method and system for introducing a gene into a human stem cell -> Monitor Keywords |
|
Method and system for introducing a gene into a human stem cellRelated Patent Categories: Chemistry: Molecular Biology And Microbiology, Process Of Mutation, Cell Fusion, Or Genetic Modification, Introduction Of A Polynucleotide Molecule Into Or Rearrangement Of Nucleic Acid Within An Animal CellMethod and system for introducing a gene into a human stem cell description/claimsThe Patent Description & Claims data below is from USPTO Patent Application 20060211113, Method and system for introducing a gene into a human stem cell. Brief Patent Description - Full Patent Description - Patent Application Claims RELATED APPLICATION [0001] This application is continuation of U.S. patent application Ser. No. 09/781,046 filed on Feb. 8, 2001, which is a continuation-in-part application claiming priority to U.S. patent application Ser. No. 09/537,861, filed on Mar. 28, 2000, both of which are hereby incorporated by reference as if fully set forth herein, including drawings. FIELD OF THE INVENTION [0002] The field of the present invention relates to vector systems for gene and stem cell therapy. In particular, it relates to a method and system for introducing a gene into a human stem cell to be used for therapeutic purposes. BACKGROUND OF THE INVENTION [0003] Various diseases originate from defective genes that are either inherited or modified during life by environmental agents. Examples of these diseases include different forms of cystic fibrosis, Tay Sachs, sickle cell anemia, Duchenne muscular dystrophy, cancer, hemophilia, or LDL receptor deficiency. In addition, certain conditions are caused by the imbalance or lack of certain hormones or growth factors in the body stemming from damage to a particular organ. For example, kidney dialysis patients are often anemic because the damaged kidneys do not produce sufficient erythropoietin to adequately stimulate production of an appropriate level of red blood cells. Gene therapy or gene replacement therapy promises, not only a treatment, but also a potential cure for these diseases by replacing defective genes or by augmenting production of certain gene products. [0004] Common vectors for introducing the therapeutic gene or nucleic acid include viral and non-viral vectors. Although viral delivery systems have been considered to be most efficient in delivering genes to cells, it may be limited because of a risk of triggering inflammatory or immunogenic responses. Forbes, S. J., "Review Article: Gene Therapy in Gastroenterology and Hepatology," Aliment Pharmacol. Ther. 11:823-826 (1997). The risk is exemplified by the death of Jesse Gelsinger, a volunteer who died on Sep. 17, 1999 while participating in a gene therapy clinical trial at the Institute for Human Gene Therapy, University of Pennsylvania. His death has fueled the controversy over the use and safety of gene therapy. The trial was directed to treat omithine transcarbmylase (OTC) using a modified adenoviral vector. The administration, however, of the vector to Gelsinger "initiated an unusual and deadly immune-system response that led to multiple organ failure and death." See Preliminary Findings, The Institute of Human Gene Therapy, University of Pennsylvania Health System, Dec. 2, 1999, <http://www.med.upenn.edu/ihgt/findings.html>. Although adenoviral vectors offer several advantages over other viral vectors in that they can infect a wide range of cells and are not limited to replicating cells, as are retroviral vectors, adenoviral vectors may activate the immune system, as seen in the Gelsinger's case, such that the initial dose or repeated introduction may become less effective, if not life threatening. See also Forbes, S. J., supra. Because other gene therapy vectors such as retrovirus or liposomes are generally foreign molecules, they similarly trigger the immune reaction and decrease the effectiveness of the therapy. [0005] In addition to gene therapy, stem-cell based therapies are promising treatments for alleviating certain diseases such as neurodegenerative diseases, hematopoetic diseases and cancers, or muscular dystrophies. For these types of diseases, stem cells that have the ability to differentiate into a specific cell types may be grown in culture and transplanted into, for example, the central nervous system, the muscles, or the bone marrow to regenerate atrophied or ablated tissues. Current protocols for stem cell-based therapies are also disadvantageous because of the potential host immune response to the graft stem cells. In addition, it is also difficult to introduce a therapeutic gene into a human stem cell if such a therapeutic gene would be desirable. [0006] Thus, there exists a need to enhance the safety and efficiency of gene therapy vectors that addresses the complexities of interacting with the immune system and the ease with which a therapeutic gene can be introduced into the body. SUMMARY OF THE INVENTION [0007] The present invention provides for a method and a vector system for delivering a polynucleotide into human stem cells for treatment or prevention of a disease or condition. In one aspect, vector system is disclosed that comprises a human sperm cell linked to a polynucleotide through a linker. The human sperm cell may be used to introduce the polynucleotide into a human oocyte during fertilization. The resulting zygote may then be cultured, differentiated, and transplanted into the human body. BRIEF DESCRIPTION OF THE DRAWINGS [0008] FIG. 1 is a pictorial representation of the basic steps involved in introducing an exogenous polynucleotide into an embryonic stem cell using a sperm cell. [0009] FIG. 2 shows a flow-cytometry result of binding a sperm-specific antibody to human sperm cells. [0010] FIG. 3 shows a flow-cytometry result of binding a sperm-specific antibody to murine sperm cells. [0011] FIG. 4 shows a flow-cytometry result of binding a sperm-specific antibody to pig's sperm cells. [0012] FIG. 5 shows a plasmid map of pCMV-.beta.. [0013] FIG. 6 show results of PCR analysis for the detection of pCMV-.beta. sequences in genomic DNA isolated from murine embryos genetically modified according to basic steps in FIG. 1. [0014] FIG. 7 shows results of southern-blot analysis for the detection of Hepatitis B surface-antigen gene-sequence in mice-tail-genomic DNA with this gene-sequence being integrated into the murine chromosome according to one embodiment of the present invention. [0015] FIG. 8 shows the plasmid map of pSEAP-2-control. [0016] FIG. 9 shows the result of southern-blot analysis for the detection of pSEAP2-control plasmid sequence in the genomic DNA isolated from tail tissues of genetically modified pigs according to one embodiment of the present invention. [0017] FIG. 10 shows the copy number of integrated pSEAP2-control plasmid in four genetically modified pigs based on densitometric intensities of bands in FIG. 13. [0018] FIGS. 11 and 12 show the results of enzyme assays for secreted alkaline phosphatase found in serum of pigs genetically modified according to one embodiment of the present invention. GENERAL DESCRIPTION OF THE PREFERRED EMBODIMENTS Continue reading about Method and system for introducing a gene into a human stem cell... Full patent description for Method and system for introducing a gene into a human stem cell Brief Patent Description - Full Patent Description - Patent Application Claims Click on the above for other options relating to this Method and system for introducing a gene into a human stem cell patent application. ### 1. Sign up (takes 30 seconds). 2. Fill in the keywords to be monitored. 3. Each week you receive an email with patent applications related to your keywords. Start now! - Receive info on patent apps like Method and system for introducing a gene into a human stem cell or other areas of interest. ### Previous Patent Application: Growth of human dendritic cells for cancer immunotherapy in closed system using microcarrier beads Next Patent Application: Modulation of hedgehog-mediated signaling pathway Industry Class: Chemistry: molecular biology and microbiology ### FreshPatents.com Support Thank you for viewing the Method and system for introducing a gene into a human stem cell patent info. IP-related news and info Results in 0.17556 seconds Other interesting Feshpatents.com categories: Canon USA , Celera Genomics , Cephalon, Inc. , Cingular Wireless , Clorox , Colgate-Palmolive , Corning , Cymer , 174 |
* Protect your Inventions * US Patent Office filing
PATENT INFO |
|