| Gene therapy clinical trials worldwide to 2017: An update SL Ginn, AK Amaya, IE Alexander, M Edelstein, MR Abedi The journal of gene medicine 20 (5), e3015, 2018 | 935 | 2018 |
| Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome GJ Logan, AP Dane, CV Hallwirth, CM Smyth, EE Wilkie, AK Amaya, ... Nature genetics 49 (8), 1267-1273, 2017 | 109 | 2017 |
| Attenuation of heparan sulfate proteoglycan binding enhances in vivo transduction of human primary hepatocytes with AAV2 M Cabanes-Creus, A Westhaus, RG Navarro, G Baltazar, E Zhu, ... Molecular Therapy Methods & Clinical Development 17, 1139-1154, 2020 | 38 | 2020 |
| Codon-optimization of wild-type adeno-associated virus capsid sequences enhances DNA family shuffling while conserving functionality M Cabanes-Creus, SL Ginn, AK Amaya, SHY Liao, A Westhaus, ... Molecular Therapy-Methods & Clinical Development 12, 71-84, 2019 | 34 | 2019 |
| Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8 M Cabanes-Creus, RG Navarro, E Zhu, G Baltazar, SHY Liao, M Drouyer, ... Molecular Therapy Methods & Clinical Development 24, 88-101, 2022 | 28 | 2022 |
| Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes SL Ginn, AK Amaya, SHY Liao, E Zhu, SC Cunningham, M Lee, ... JHEP Reports 2 (1), 100065, 2020 | 26 | 2020 |
| Gene therapy: paving new roads in the treatment of hemophilia GG Yamaguti-Hayakawa, MC Ozelo Seminars in thrombosis and hemostasis 45 (07), 743-750, 2019 | 20 | 2019 |
| Assessment of pre-clinical liver models based on their ability to predict the liver-tropism of adeno-associated virus vectors A Westhaus, M Cabanes-Creus, KL Dilworth, E Zhu, D Salas Gomez, ... Human Gene Therapy 34 (7-8), 273-288, 2023 | 12 | 2023 |
| Gene therapy clinical trials worldwide to 2017: An update (vol 20, e3015, 2018) SL Ginn, AK Amaya, IE Alexander, M Edelstein, MR Abedi JOURNAL OF GENE MEDICINE 21 (9), 2019 | 2 | 2019 |
| Secreted Particle Information Transfer (SPIT)–A Cellular Platform for In Vivo Genetic Engineering CT Charlesworth, S Homma, F Suchy, S Wang, J Bhadhury, AK Amaya, ... bioRxiv, 2024 | | 2024 |
| Assessment of pre-clinical liver models based on their ability to predict the liver-tropism of AAV vectors A Westhaus, M Cabanes-Creus, KL Dilworth, E Zhu, DS Gómez, ... bioRxiv, 2022.09. 28.510021, 2022 | | 2022 |
| Inhibition of proliferation in primary human hepatocytes following in vivo AAV-mediated genome editing AK Amaya, SL Ginn, SHY Liao, C Zhu, CM Smyth, GJ Logan, L Lisowski, ... HUMAN GENE THERAPY 30 (11), A18-A18, 2019 | | 2019 |
| Efficient in vivo editing of patient-derived primary human hepatocytes SL Ginn, AK Amaya, SHY Liao, E Zhu, SC Cunningham, CV Hallwirth, ... HUMAN GENE THERAPY 30 (8), A2-A3, 2019 | | 2019 |
| Towards the Treatment of Human Genetic Liver Disease by AAV-Mediated Genome Editing and Selective Expansion of Repaired Hepatocytes AK Amaya Colina | | 2019 |
| Successful in vivo editing of patient-derived primary human hepatocytes AK Amaya, SL Ginn, SHY Liao, C Zhu, M Lee, HA Pickett, CV Hallwirth, ... HUMAN GENE THERAPY 29 (12), A26-A27, 2018 | | 2018 |
| Changing the Face of Modern Medicine: Stem Cell and Gene Therapy Organized Jointly by the European Society of Gene & Cell Therapy (ESGCT), International Society for Stem Cell … KM Champion, P Albertini, I Visigalli, F Cecere, N Carriglio, ... Human Gene Therapy 29 (12), A1-A169, 2018 | | 2018 |
| Successful In Vivo Editing of the OTC Locus in Primary Human Hepatocytes Xenografted into the FRG Mouse Liver AK Amaya, SL Ginn, SHY Liao, C Zhu, M Lee, HA Pickett, CV Hallwirth, ... MOLECULAR THERAPY 26 (5), 448-449, 2018 | | 2018 |
| Evaluation of Recombinant Adeno-Associated Virus-Based Genome Editing Reagents for Homology-Directed Repair to Target a Human Liver Locus In Vivo SL Ginn, AK Amaya, SHY Liao, C Zhu, CV Hallwirth, SS Tay, ... MOLECULAR THERAPY 26 (5), 113-113, 2018 | | 2018 |
| TREATMENT OF GENETIC LIVER DISEASE BY AAV-MEDIATED GENOME EDITING AND SELECTIVE EXPANSION OF REPAIRED HEPATOCYTES AK Amaya, C Zhu, SL Ginn, CV Hallwirth, SS Tay, SC Cunningham, ... JOURNAL OF GENE MEDICINE 20 (1), 2018 | | 2018 |
| DISCOVERY OF A LIVER-SPECIFIC ENHANCER-PROMOTER ELEMENT IN THE 3'UTR OF THE WILD-TYPE AAV2 GENOME PROVIDES NOVEL INSIGHTS INTO AAV VECTOR SAFETY IN THE HUMAN LIVER GJ Logan, AP Dane, CV Hallwirth, CM Smyth, EE Wilkie, AK Amaya, ... JOURNAL OF GENE MEDICINE 20 (1), 2018 | | 2018 |
