http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2019203225-A1

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inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_14b88de0f046d97e8ff34f1f2163f208
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publicationDate 2019-07-04-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2019203225-A1
titleOfInvention Gene therapy for patients with fanconi anemia
abstract The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed.
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