IN VIVO GENE ENGINEERING WITH ADENOVIRAL VECTORS

The present invention provides recombinant nucleic acid expression cassetie and helper dependent adenovirus, where the expression cassettes utilize a miRNA based system for controlling expression of nucleases in helper dependent adenoviral viral producer cells, thus permitting production and use for...

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Bibliographic Details
Main Authors RICHTER, Maximilian, PAPAYANNOPOULOU, Thalia, LIEBER, Andre, SAYDAMINOVA, Kamola
Format Patent
LanguageEnglish
French
German
Published 08.03.2017
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary:The present invention provides recombinant nucleic acid expression cassetie and helper dependent adenovirus, where the expression cassettes utilize a miRNA based system for controlling expression of nucleases in helper dependent adenoviral viral producer cells, thus permitting production and use for in in vivo gene editing in CD34+ cells.
Bibliography:Application Number: EP20150785973