| Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice A Cantore, N Nair, P Della Valle, M Di Matteo, J Matrai, F Sanvito, ... Blood, The Journal of the American Society of Hematology 120 (23), 4517-4520, 2012 | 105 | 2012 |
| Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells S Dastidar, S Ardui, K Singh, D Majumdar, N Nair, Y Fu, D Reyon, ... Nucleic acids research 46 (16), 8275-8298, 2018 | 89 | 2018 |
| Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates MK Chuah, I Petrus, P De Bleser, C Le Guiner, G Gernoux, O Adjali, ... Molecular Therapy 22 (9), 1605-1613, 2014 | 86 | 2014 |
| Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy N Nair, MY Rincon, H Evens, S Sarcar, S Dastidar, E Samara-Kuko, ... Blood, The Journal of the American Society of Hematology 123 (20), 3195-3199, 2014 | 86 | 2014 |
| Efficient in vivo liver-directed gene editing using CRISPR/Cas9 K Singh, H Evens, N Nair, MY Rincón, S Sarcar, E Samara-Kuko, ... Molecular Therapy 26 (5), 1241-1254, 2018 | 76 | 2018 |
| Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc‐/‐mice C Levy, F Fusil, F Amirache, C Costa, A Girard‐Gagnepain, D Negre, ... Journal of Thrombosis and Haemostasis 14 (12), 2478-2492, 2016 | 60 | 2016 |
| AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model A Verhelle, N Nair, I Everaert, W Van Overbeke, L Supply, O Zwaenepoel, ... Human molecular genetics 26 (7), 1353-1364, 2017 | 49 | 2017 |
| Recent progress in gene therapy for hemophilia MK Chuah, N Nair, T VandenDriessche Human gene therapy 23 (6), 557-565, 2012 | 45 | 2012 |
| piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts M Loperfido, S Jarmin, S Dastidar, M Di Matteo, I Perini, M Moore, N Nair, ... Nucleic acids research 44 (2), 744-760, 2016 | 35 | 2016 |
| FANCA knockout in human embryonic stem cells causes a severe growth disadvantage K Vanuytsel, Q Cai, N Nair, S Khurana, S Shetty, JR Vermeesch, ... Stem Cell Research 13 (2), 240-250, 2014 | 13 | 2014 |
| ESGCT XXV anniversary congress in collaboration with the German Society for Gene Therapy, October 17–20, 2017 Berlin, Germany S Maffioletti Human Gene Therapy 28 (12), A1-A125, 2017 | 2 | 2017 |
| Computationally designed liver-specific transcriptional cis-regulatory modules and hyper-functional factor IX improve liver-targeted gene therapy for hemophilia B. NNS Nair, MY Rincon, H Evens, S Sarcar, S Dastidar, E Samara, ... Blood 123 (20), 3195-3199, 2014 | 2 | 2014 |
| Solving the T-cell problem: generation of immune stealth AAV T VandenDriessche, F Mingozzi, NNS Nair, J Willems, H Evens, ... Hum Gene Ther 23 (10), A23-A23, 2012 | 2 | 2012 |
| European society of gene and cell therapy french society of cell and gene therapy collaborative congress 2012 october 25–29, 2012 palais des congrès de versailles, France T VandenDriessche, RA Pearson, RJ Chandler, CC Bartholomae, ... Hum. Gene Ther 23 (10), 2012 | 2 | 2012 |
| AAV BASED HEMOPHILIA B GENE THERAPY IN MICE USING FIX-CB2679d-GT GE Blouse, N Nair, T VandenDriessche, MK Chuah, J Landau | 1 | 2019 |
| CRISPR/Cas9-mediated editing for dominant genetic disorders: efficient excision of trinucleotide repeat expansion in myotonic dystrophy S Dastidar, S Ardui, K Singh, N Nair, Y Fu, D Reyon, E Samara, MFM Gerli, ... Human Gene Therapy 28 (12), A7-A7, 2017 | 1 | 2017 |
| Gene therapy for hemophilia A and B N Nair, M Chuah, T VandenDriessche Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology …, 2020 | | 2020 |
| Truncated gRNAs result in efficient in vivo liver-directed gene inactivation using CRISPR/Cas9 K Singh, H Evens, N Nair, MY Rincon, S Sarcar, E Samara, MK Chuah, ... Human Gene Therapy 29 (12), A112-A112, 2018 | | 2018 |
| CRISPR/Cas9-Mediated Editing of Trinucleotide Repeat Expansion in Myotonic Dystrophy S Dastidar, K Singh, N Nair, Y Fu, D Reyon, E Samara, MFM Gerli, ... Molecular Therapy 25 (5), 85-85, 2017 | | 2017 |
| 120. Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9 K Singh, H Evens, M Rincón, N Nair, S Sarcar, E Samara-Kuko, ... Molecular Therapy 24, S50, 2016 | | 2016 |
Melvin Yesid Rincon AcelasM.D., Ph.D., Principal Scientist Gene Therapy UCBVerified email at ucb.com
