| Efficacy and safety of sirolimus in lymphangioleiomyomatosis FX McCormack, Y Inoue, J Moss, LG Singer, C Strange, K Nakata, ... New England Journal of Medicine 364 (17), 1595-1606, 2011 | 1148 | 2011 |
| Molecular basis of alpha-1-antitrypsin deficiency M Brantly, T Nukiwa, RG Crystal The American journal of medicine 84, 13-31, 1988 | 697 | 1988 |
| Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I K Overturf, M Al-Dhalimy, R Tanguay, M Brantly, CN Ou, M Finegold, ... Nature genetics 12 (3), 266-273, 1996 | 630 | 1996 |
| Clinical features and history of the destructive lung disease associated with alpha-1-antitrypsin deficiency of adults with pulmonary symptoms ML Brantly, LD Paul, BH Miller, RT Falk, M Wu, RG Crystal Am Rev Respir Dis 138 (2), 327-336, 1988 | 406 | 1988 |
| Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results TR Flotte, BC Trapnell, M Humphries, B Carey, R Calcedo, F Rouhani, ... Human gene therapy 22 (10), 1239-1247, 2011 | 379 | 2011 |
| Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy ML Brantly, JD Chulay, L Wang, C Mueller, M Humphries, LT Spencer, ... Proceedings of the National Academy of Sciences 106 (38), 16363-16368, 2009 | 376 | 2009 |
| Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors S Song, M Morgan, T Ellis, A Poirier, K Chesnut, J Wang, M Brantly, ... Proceedings of the National Academy of Sciences 95 (24), 14384-14388, 1998 | 356 | 1998 |
| American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency. JK Stoller, GL Snider, ML Brantly, RJ Fallat, RA Stockley, GM Turino, ... American Journal of Respiratory and Critical Care Medicine 168, 818-900, 2003 | 342 | 2003 |
| Genetic defects and clinical characteristics of patients with a form of oculocutaneous albinism (Hermansky–Pudlak syndrome) WA Gahl, M Brantly, MI Kaiser-Kupfer, F Iwata, S Hazelwood, ... New England Journal of Medicine 338 (18), 1258-1265, 1998 | 333 | 1998 |
| Baseline characteristics of enrollees in the National Heart, Lung and Blood Institute Registry of alpha1-antitrypsin deficiency NG McElvaney, JK Stoller, AS Buist, UBS Prakash Chest 111 (2), 394, 1997 | 286 | 1997 |
| α1-Antitrypsin deficiency CM Greene, SJ Marciniak, J Teckman, I Ferrarotti, ML Brantly, DA Lomas, ... Nature reviews Disease primers 2 (1), 1-17, 2016 | 262 | 2016 |
| Rapid, nonradioactive detection of mutations in the human genome by allele-specific amplification H Okayama, DT Curiel, ML Brantly, MD Holmes, RG Crystal The Journal of laboratory and clinical medicine 114 (2), 105-113, 1989 | 253 | 1989 |
| Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults TR Flotte, ML Brantly, LT Spencer, BJ Byrne, CT Spencer, DJ Baker, ... Human gene therapy 15 (1), 93-128, 2004 | 229 | 2004 |
| Clinical features of individuals with PI* SZ phenotype of alpha 1-antitrypsin deficiency. alpha 1-Antitrypsin Deficiency Registry Study Group. GM Turino, AF Barker, ML Brantly, AB Cohen, RP Connelly, RG Crystal, ... American journal of respiratory and critical care medicine 154 (6), 1718-1725, 1996 | 225 | 1996 |
| Use of a highly purified α1-antitrypsin standard to establish ranges for the common normal and deficient α1-antitrypsin phenotypes ML Brantly, JT Wittes, CF Vogelmeier, RC Hubbard, GA Fells, RG Crystal Chest 100 (3), 703-708, 1991 | 223 | 1991 |
| The alpha1-antitrypsin gene and its mutations: Clinical consequences and strategies for therapy RG Crystal, ML Brantly, RC Hubbard, DT Curiel, MD Holmes Chest 95 (1), 196-208, 1989 | 222 | 1989 |
| Effect of pirfenidone on the pulmonary fibrosis of Hermansky–Pudlak syndrome WA Gahl, M Brantly, J Troendle, NA Avila, A Padua, C Montalvo, ... Molecular genetics and metabolism 76 (3), 234-242, 2002 | 220 | 2002 |
| Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2 α1-Antitrypsin (AAT) Vector in AAT-Deficient Adults ML Brantly, LT Spencer, M Humphries, TJ Conlon, CT Spencer, A Poirier, ... Human gene therapy 17 (12), 1177-1186, 2006 | 217 | 2006 |
| The diagnosis and management of alpha-1 antitrypsin deficiency in the adult RA Sandhaus, G Turino, ML Brantly, M Campos, CE Cross, K Goodman, ... Chronic Obstructive Pulmonary Diseases: Journal of the COPD Foundation 3 (3 …, 2016 | 212 | 2016 |
| Z α1-antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant AT Mulgrew, CC Taggart, MW Lawless, CM Greene, ML Brantly, ... Chest 125 (5), 1952-1957, 2004 | 202 | 2004 |
