http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CA-2488249-A1
Outgoing Links
Predicate | Object |
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assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_f07ba5e7ff356b7eda43fdfc20eef4b0 http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_d843b58d53ccd702ee67df059e0f70bf |
classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2510-02 |
classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-005 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N5-0688 |
classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-7088 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-09 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-071 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 |
filingDate | 2003-06-05-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f6fa12fc91e4e711ccf6185871c8f484 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_14feea0511b6c59e751dae5454aa5a78 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ff3a3acbb6dbd9e001ecf6d3cec2e7d5 |
publicationDate | 2003-12-18-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber | CA-2488249-A1 |
titleOfInvention | Spliceosome mediated rna trans-splicing in stem cells |
abstract | The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated trans-splicing in stem cells. The compositions of the invention include stem cells engineered to express pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans~-splicing reaction resulting in the generation of novel chimeric RNA molecules (chimeric RNA). In particular, the stem cells of the present invention are genetically engineered to express a PTM that will interact with a specific target pre-mRNA expressed within a stem cell as it differentiates so as to result in correction of a genetic defect responsible for a genetic disorder. The methods of the invention encompass transferring a nucleic acid molecule capable of encoding a PTM of interest into a stem cell followed by transplantation of the PTM modified stem cell into a host. As the stem cell differentiates the target pre-mRNA is expressed thereby providing the substrate for a trans-splicing reaction. The present invention is based on the successful transfer and expression of a nucleic acid molecule encoding a PTM capable of interacting with a cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA into primary human surface airway progenitor cells. The methods and compositions of the present invention can be used to correct genetic defects associated with a variety of different disorders such as cystic fibrosis, hemophilia, sickle cell anemia, Tay-Sachs disease, thalassemias, polycystic kidney disease and muscular dystrophy, to name a few. |
priorityDate | 2002-06-05-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 623.