Carmen C. Brewer

Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen

Laura A. Johnson, Richard A. Morgan, Mark E. Dudley et al. · Blood · 2009 · 1.4K citations · Full text

Neonatal-Onset Multisystem Inflammatory Disease Responsive to Interleukin-1β Inhibition

Raphaela Goldbach‐Mansky, Natalie Dailey Garnes, Scott Canna et al. · New England Journal of Medicine · 2006 · 888 citations · Full text

Phenotype and Course of Hutchinson–Gilford Progeria Syndrome

Melissa A. Merideth, Leslie B. Gordon, Sarah Clauss et al. · New England Journal of Medicine · 2008 · 734 citations · Full text

GATA2 deficiency: a protean disorder of hematopoiesis, lymphatics, and immunity

Michael A. Spinner, Lauren Sanchez, Amy P. Hsu et al. · Blood · 2013 · 691 citations · Full text

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

Daniel S. Ory, Elizabeth A. Ottinger, Nicole Y. Farhat et al. · The Lancet · 2017 · 316 citations · Full text

Neurodegenerative Diseases, Neurobiology Of Disease, Clinical Therapeutic +9