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Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells.
Ferrari S, Jacob A, Cesana D, Laugel M, Beretta S, Varesi A, Unali G, Conti A, Canarutto D, Albano L, Calabria A, Vavassori V, Cipriani C, Castiello MC, Esposito S, Brombin C, Cugnata F, Adjali O, Ayuso E, Merelli I, Villa A, Di Micco R, Kajaste-Rudnitski A, Montini E, Penaud-Budloo M, Naldini L. Ferrari S, et al. Among authors: ayuso e. Cell Stem Cell. 2022 Oct 6;29(10):1428-1444.e9. doi: 10.1016/j.stem.2022.09.001. Cell Stem Cell. 2022. PMID: 36206730 Free PMC article.
Optimized delivery of integrase-defective lentiviral vector (IDLV) induced lower DNA load and less persistent DDR, improving clonogenic capacity and editing efficiency in long-term repopulating HSPCs. Because insertions of viral DNA fragments are less frequent with IDLV, i …
Optimized delivery of integrase-defective lentiviral vector (IDLV) induced lower DNA load and less persistent DDR, improving clonogenic capa …
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates.
Milani M, Canepari C, Liu T, Biffi M, Russo F, Plati T, Curto R, Patarroyo-White S, Drager D, Visigalli I, Brombin C, Albertini P, Follenzi A, Ayuso E, Mueller C, Annoni A, Naldini L, Cantore A. Milani M, et al. Among authors: ayuso e. Nat Commun. 2022 May 4;13(1):2454. doi: 10.1038/s41467-022-30102-3. Nat Commun. 2022. PMID: 35508619 Free PMC article.
Stable nearly life-long normal and above-normal factor VIII activity was achieved in hemophilia A mouse models. Overall, we show long-term factor VIII activity and restoration of hemostasis, by lentiviral gene therapy to hemophilia A mice and normal-range factor VIII activ …
Stable nearly life-long normal and above-normal factor VIII activity was achieved in hemophilia A mouse models. Overall, we show long-ter …
Intrinsic Differential Scanning Fluorimetry for Fast and Easy Identification of Adeno-Associated Virus Serotypes.
Rieser R, Penaud-Budloo M, Bouzelha M, Rossi A, Menzen T, Biel M, Büning H, Ayuso E, Winter G, Michalakis S. Rieser R, et al. Among authors: ayuso e. J Pharm Sci. 2020 Jan;109(1):854-862. doi: 10.1016/j.xphs.2019.10.031. Epub 2019 Oct 19. J Pharm Sci. 2020. PMID: 31639391
Recombinant adeno-associated virus (AAV) vectors have evolved as the most promising technology for gene therapy due to their good safety profile, high transduction efficacy, and long-term gene expression in non-dividing cells. AAV-based gene therapy holds great promise for …
Recombinant adeno-associated virus (AAV) vectors have evolved as the most promising technology for gene therapy due to their good safety pro …
Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs.
Jaén ML, Vilà L, Elias I, Jimenez V, Rodó J, Maggioni L, Ruiz-de Gopegui R, Garcia M, Muñoz S, Callejas D, Ayuso E, Ferré T, Grifoll I, Andaluz A, Ruberte J, Haurigot V, Bosch F. Jaén ML, et al. Among authors: ayuso e. Mol Ther Methods Clin Dev. 2017 Apr 5;6:1-7. doi: 10.1016/j.omtm.2017.03.008. eCollection 2017 Sep 15. Mol Ther Methods Clin Dev. 2017. PMID: 28626777 Free PMC article.
Previous studies proved the feasibility of gene delivery to large diabetic animals with adeno-associated viral (AAV) vectors. Here, we report the long-term (8 years) follow-up after a single administration of therapeutic vectors to diabetic dogs. ...The persistence of vect …
Previous studies proved the feasibility of gene delivery to large diabetic animals with adeno-associated viral (AAV) vectors. Here, we repor …
Pancreatic transduction by helper-dependent adenoviral vectors via intraductal delivery.
Morró M, Teichenne J, Jimenez V, Kratzer R, Marletta S, Maggioni L, Mallol C, Ruberte J, Kochanek S, Bosch F, Ayuso E. Morró M, et al. Among authors: ayuso e. Hum Gene Ther. 2014 Sep;25(9):824-36. doi: 10.1089/hum.2013.182. Hum Gene Ther. 2014. PMID: 25046147 Free PMC article.
Helper-dependent adenoviral vectors (HDAds) are promising tools for gene therapy because of their large cloning capacity, high levels of transgene expression, and long-term persistence in immunocompetent animals. Nevertheless, the ability of HDAds to transduce the pancreas …
Helper-dependent adenoviral vectors (HDAds) are promising tools for gene therapy because of their large cloning capacity, high levels of tra …
Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.
Callejas D, Mann CJ, Ayuso E, Lage R, Grifoll I, Roca C, Andaluz A, Ruiz-de Gopegui R, Montané J, Muñoz S, Ferre T, Haurigot V, Zhou S, Ruberte J, Mingozzi F, High KA, Garcia F, Bosch F. Callejas D, et al. Among authors: ayuso e. Diabetes. 2013 May;62(5):1718-29. doi: 10.2337/db12-1113. Epub 2013 Feb 1. Diabetes. 2013. PMID: 23378612 Free PMC article.
We previously demonstrated that it is possible to generate a "glucose sensor" in skeletal muscle through coexpression of glucokinase and insulin, increasing glucose uptake and correcting hyperglycemia in diabetic mice. Here, we demonstrate long-term efficacy of this approa …
We previously demonstrated that it is possible to generate a "glucose sensor" in skeletal muscle through coexpression of glucokinase and ins …
In vivo genetic engineering of murine pancreatic beta cells mediated by single-stranded adeno-associated viral vectors of serotypes 6, 8 and 9.
Jimenez V, Ayuso E, Mallol C, Agudo J, Casellas A, Obach M, Muñoz S, Salavert A, Bosch F. Jimenez V, et al. Among authors: ayuso e. Diabetologia. 2011 May;54(5):1075-86. doi: 10.1007/s00125-011-2070-3. Epub 2011 Feb 11. Diabetologia. 2011. PMID: 21311856
AAV9 vectors encoding hepatocyte growth factor (HGF) were delivered intraductally to a transgenic mouse model of type 1 diabetes and glycaemia was monitored. RESULTS: AAV6, AAV8 and AAV9 mediated efficient and long-term transduction of beta cells, with AAV6 and AAV8 showin …
AAV9 vectors encoding hepatocyte growth factor (HGF) were delivered intraductally to a transgenic mouse model of type 1 diabetes and glycaem …
Increased ocular levels of IGF-1 in transgenic mice lead to diabetes-like eye disease.
Ruberte J, Ayuso E, Navarro M, Carretero A, Nacher V, Haurigot V, George M, Llombart C, Casellas A, Costa C, Bosch A, Bosch F. Ruberte J, et al. Among authors: ayuso e. J Clin Invest. 2004 Apr;113(8):1149-57. doi: 10.1172/JCI19478. J Clin Invest. 2004. PMID: 15085194 Free PMC article.
These findings suggest a role of IGF-1 in the development of ocular complications in long-term diabetes. Thus, these transgenic mice may be used to study the mechanisms that lead to diabetes eye disease and constitute an appropriate model in which to assay new therapies... …
These findings suggest a role of IGF-1 in the development of ocular complications in long-term diabetes. Thus, these transgenic mice …