Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene

β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-gl...

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Publicat a:Hum Gene Ther
Autors principals: Negre, Olivier, Eggimann, Anne-Virginie, Beuzard, Yves, Ribeil, Jean-Antoine, Bourget, Philippe, Borwornpinyo, Suparerk, Hongeng, Suradej, Hacein-Bey, Salima, Cavazzana, Marina, Leboulch, Philippe, Payen, Emmanuel
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2016
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Reviews
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4779296/
https://ncbi.nlm.nih.gov/pubmed/26886832
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.007
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