| Selection and evaluation of clinically relevant AAV variants in a xenograft liver model L Lisowski, AP Dane, K Chu, Y Zhang, SC Cunningham, EM Wilson, ... Nature 506 (7488), 382-386, 2014 | 459 | 2014 |
| Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa V Sebastiano, HH Zhen, B Haddad, E Bashkirova, SP Melo, P Wang, ... Science translational medicine 6 (264), 264ra163-264ra163, 2014 | 221 | 2014 |
| Genome editing of isogenic human induced pluripotent stem cells recapitulates long QT phenotype for drug testing Y Wang, P Liang, F Lan, H Wu, L Lisowski, M Gu, S Hu, MA Kay, ... Journal of the American College of Cardiology 64 (5), 451-459, 2014 | 175 | 2014 |
| Bioengineered AAV capsids with combined high human liver transduction in vivo and unique humoral seroreactivity Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP ... Molecular Therapy 26 (1), 289-303, 2018 | 173 | 2018 |
| Adeno-associated virus serotypes for gene therapeutics L Lisowski, SS Tay, IE Alexander Current opinion in pharmacology 24, 59-67, 2015 | 166 | 2015 |
| A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference S Samakoglu, L Lisowski, T Budak-Alpdogan, Y Usachenko, S Acuto, ... Nature biotechnology 24 (1), 89-94, 2006 | 161 | 2006 |
| In situ gene therapy via AAV-CRISPR-Cas9-mediated targeted gene regulation AM Moreno, X Fu, J Zhu, D Katrekar, YRV Shih, J Marlett, J Cabotaje, ... Molecular Therapy 26 (7), 1818-1827, 2018 | 149 | 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 | 106 | 2017 |
| Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging Y Wang, WY Zhang, S Hu, F Lan, AS Lee, B Huber, L Lisowski, P Liang, ... Circulation research 111 (12), 1494-1503, 2012 | 102 | 2012 |
| Optogenetic approaches to vision restoration MP Simunovic, W Shen, JY Lin, DA Protti, L Lisowski, MC Gillies Experimental eye research 178, 15-26, 2019 | 101 | 2019 |
| An atypical parvovirus drives chronic tubulointerstitial nephropathy and kidney fibrosis B Roediger, Q Lee, S Tikoo, JCA Cobbin, JM Henderson, M Jormakka, ... Cell 175 (2), 530-543. e24, 2018 | 100 | 2018 |
| Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity WMM Winters IP, Chiou SH, Paulk NK, McFarland CD, Lalgudi PV, Ma RK ... Nature Communications 8 (1), 2053, 2017 | 97 | 2017 |
| The balance of stromal BMP signaling mediated by GREM1 and ISLR drives colorectal carcinogenesis H Kobayashi, KA Gieniec, JA Wright, T Wang, N Asai, Y Mizutani, T Lida, ... Gastroenterology 160 (4), 1224-1239. e30, 2021 | 90 | 2021 |
| High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction A Westhaus, M Cabanes-Creus, A Rybicki, G Baltazar, RG Navarro, E Zhu, ... Human gene therapy 31 (9-10), 575-589, 2020 | 78 | 2020 |
| Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in β-thalassemic mice L Lisowski, M Sadelain Blood, The Journal of the American Society of Hematology 110 (13), 4175-4178, 2007 | 73 | 2007 |
| Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant SP Melo, L Lisowski, E Bashkirova, HH Zhen, K Chu, DR Keene, ... Molecular Therapy 22 (4), 725-733, 2014 | 71 | 2014 |
| Age-related seroprevalence of antibodies against AAV-LK03 in a UK population cohort DP Perocheau, SC Cunningham, J Lee, J Antinao Diaz, SN Waddington, ... Human gene therapy 30 (1), 79-87, 2019 | 61 | 2019 |
| Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing MT Nguyen Tran, MKN Mohd Khalid, Q Wang, JKR Walker, ... Nature Communications 11 (1), 4871, 2020 | 49 | 2020 |
| A user's guide to the inverted terminal repeats of adeno-associated virus P Wilmott, L Lisowski, IE Alexander, GJ Logan Human gene therapy methods 30 (6), 206-213, 2019 | 48 | 2019 |
| AAV capsid proteins for nucleic acid transfer L Lisowski, MA Kay US Patent 9,169,299, 2015 | 48 | 2015 |
