Title: | Long-term safety and efficacy of factor IX gene therapy in hemophilia B |
Journal : | The New England journal of medicine, 371, 21 |
Authors: | Nathwani AC, Author ; Reiss UM ; Tuddenham EG ; Rosales C ; Chowdary P ; McIntosh J ; Della Peruta M ; Lheriteau E ; Patel N ; Raj D ; Riddell A ; Pie J ; Rangarajan S ; Bevan D ; Recht M ; Shen YM ; Halka KG ; Basner-Tschakarjan E ; Mingozzi F ; High KA ; Allay J ; Kay MA ; Ng CY ; Zhou J ; Cancio M ; Morton CL ; Gray JT ; Srivastava D ; Nienhuis AW ; Davidoff AM |
Material Type: | Article |
Publication Date: | 2014 |
Size: | p 1994 |
Languages: | English |
Abstract: |
Comment in:
Moving forward toward a cure for hemophilia B. [Mol Ther. 2015] Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice. [Gene Ther. 2016] |
Pubmed / DOI : | DOI : 10.1056/NEJMoa1407309 / Pubmed : 25409372 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/25409372 |