Gene therapy 2017: progress and future directions AM Keeler, MK ElMallah, TR Flotte Clinical and translational science 10 (4), 242, 2017 | 185 | 2017 |
Amblyopia: is visual loss permanent? MK El Mallah, U Chakravarthy, PM Hart British Journal of Ophthalmology 84 (9), 952-956, 2000 | 176 | 2000 |
Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1G93A Mice and Nonhuman Primates F Borel, G Gernoux, B Cardozo, JP Metterville, G T. Cabrera, L Song, ... Human gene therapy 27 (1), 19-31, 2016 | 135 | 2016 |
The respiratory neuromuscular system in Pompe disease DD Fuller, MK ElMallah, BK Smith, M Corti, LA Lawson, DJ Falk, BJ Byrne Respiratory physiology & neurobiology 189 (2), 241-249, 2013 | 132 | 2013 |
Pompe disease gene therapy BJ Byrne, DJ Falk, CA Pacak, S Nayak, RW Herzog, ME Elder, SW Collins, ... Human molecular genetics 20 (R1), R61-R68, 2011 | 129 | 2011 |
Adeno‐associated virus–delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model L Stoica, SH Todeasa, GT Cabrera, JS Salameh, MK ElMallah, C Mueller, ... Annals of neurology 79 (4), 687-700, 2016 | 116 | 2016 |
Peripheral nerve and neuromuscular junction pathology in Pompe disease DJ Falk, AG Todd, S Lee, MS Soustek, MK ElMallah, DD Fuller, ... Human molecular genetics 24 (3), 625-636, 2015 | 98 | 2015 |
Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice MK ElMallah, DJ Falk, S Nayak, RA Federico, MS Sandhu, A Poirier, ... Molecular Therapy 22 (4), 702-712, 2014 | 98 | 2014 |
Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema F Borel, H Sun, M Zieger, A Cox, B Cardozo, W Li, G Oliveira, A Davis, ... Proceedings of the National Academy of Sciences 115 (11), 2788-2793, 2018 | 89 | 2018 |
Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease DJ Falk, CS Mah, MS Soustek, KZ Lee, MK ElMallah, DA Cloutier, ... Molecular Therapy 21 (9), 1661-1667, 2013 | 76 | 2013 |
Hypoglossal neuropathology and respiratory activity in pompe mice KZ Lee, K Qiu, MS Sandhu, MK Elmallah, DJ Falk, MA Lane, PJ Reier, ... Frontiers in physiology 2, 31, 2011 | 56 | 2011 |
Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature AA Khan, LE Case, M Herbert, S DeArmey, H Jones, K Crisp, ... Genetics in Medicine 22 (5), 898-907, 2020 | 54 | 2020 |
Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9 MK ElMallah, DJ Falk, MA Lane, TJ Conlon, KZ Lee, NI Shafi, PJ Reier, ... Human Gene Therapy, Part B: Methods 23 (2), 148-156, 2012 | 54 | 2012 |
Development of rAAV2-CFTR: history of the first rAAV vector product to be used in humans HS Loring, MK ElMallah, TR Flotte Human gene therapy methods 27 (2), 49-58, 2016 | 53 | 2016 |
Neuropathology in respiratory-related motoneurons in young Pompe (Gaa−/−) mice SMF Turner, AK Hoyt, MK ElMallah, DJ Falk, BJ Byrne, DD Fuller Respiratory physiology & neurobiology 227, 48-55, 2016 | 51 | 2016 |
Stimulation of respiratory motor output and ventilation in a murine model of Pompe disease by ampakines MK ElMallah, S Pagliardini, SM Turner, AJ Cerreta, DJ Falk, BJ Byrne, ... American journal of respiratory cell and molecular biology 53 (3), 326-335, 2015 | 51 | 2015 |
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing TJ Gonzalez, KE Simon, LO Blondel, MM Fanous, AL Roger, ... Nature communications 13 (1), 5947, 2022 | 43 | 2022 |
Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease AM Keeler, M Zieger, SH Todeasa, AL McCall, JC Gifford, S Birsak, ... Human Gene Therapy 30 (1), 57-68, 2019 | 43 | 2019 |
Management of patients with cystic fibrosis and allergic bronchopulmonary aspergillosis using anti-immunoglobulin E therapy (omalizumab) MK ElMallah, L Hendeles, RG Hamilton, C Capen, PM Schuler The Journal of Pediatric Pharmacology and Therapeutics 17 (1), 88-92, 2012 | 43 | 2012 |
Pediatric obstructive sleep apnea in high-risk populations: clinical implications ME Mallah, E Bailey, M Trivedi, T Kremer, LM Rhein Pediatric annals 46 (9), e336-e339, 2017 | 39 | 2017 |