Collagen VI Glycine Mutations: Perturbed Assembly and a Spectrum of Clinical Severity

Por Pace, Rishika A., Peat, Rachel A., Baker, Naomi L., Zamurs, Laura, Mörgelin, Matthias, Irving, Melita, Adams, Naomi E., Bateman, John F., Mowat, David, Smith, Nicholas J. C., Lamont, Phillipa J., Moore, Steven A., Mathews, Katherine D., North, Kathryn N., Lamandé, Shireen R.
Publicado em 2008

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Por Eichler, Florian, Duncan, Christine, Musolino, Patricia L., Orchard, Paul J., De Oliveira, Satiro, Thrasher, Adrian J., Armant, Myriam, Dansereau, Colleen, Lund, Troy C., Miller, Weston P., Raymond, Gerald V., Sankar, Raman, Shah, Ami J., Sevin, Caroline, Gaspar, H. Bobby, Gissen, Paul, Amartino, Hernan, Bratkovic, Drago, Smith, Nicholas J.C., Paker, Asif M., Shamir, Esther, O’Meara, Tara, Davidson, David, Aubourg, Patrick, Williams, David A.
Publicado no N Engl J Med (2017)