In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

Autor: Shangaris, Panicos, Loukogeorgakis, Stavros P., Subramaniam, Sindhu, Flouri, Christina, Jackson, Laurence H., Wang, Wei, Blundell, Michael P., Liu, Shanrun, Eaton, Simon, Bakhamis, Nahla, Ramachandra, Durrgah Latchumi, Maghsoudlou, Panayiotis, Urbani, Luca, Waddington, Simon N., Eddaoudi, Ayad, Archer, Joy, Antoniou, Michael N., Stuckey, Daniel J., Schmidt, Manfred, Thrasher, Adrian J., Ryan, Thomas M., Coppi, Paolo De, David, Anna L.

Publikováno v: Scientific Reports, Vol 9, Iss 1, Pp 1-17 (2019)
Scientific Reports

In utero gene therapy (IUGT) to the fetal hematopoietic compartment could be used to treat congenital blood disorders such as β-thalassemia. A humanised mouse model of β-thalassemia was used, in which heterozygous animals are anaemic with splenomeg

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::1aee0133cdf65fc5b6bcf11fa510f3ff
https://lirias.kuleuven.be/handle/20.500.12942/692360

Correction of Hemoglobin Levels in a Heterozygous Humanized Mouse Model of Thalassemia after Fetal Gene Therapy

Autor: Shangaris, Panicos ^*, Loukogeorgakis, Stavros P ^*, Subramaniam, Sindhu ^*, Blundell, Mike ^*, Bakhamis, Nahla ^*, Liu, Shanrun ^*, Eaton, Simon ^*, Ramachandra, Durrgah ^*, Maghsoudlou, Panayiotis ^*, Urbani, Luca ^*, Waddington, Simon ^*, Archer, Joy ^*, Antoniou, Mike ^*, Thrasher, Adrian J ^*, Ryan, Thomas ^*, De Coppi, Paolo ^*, David, Anna ^*

Publikováno v: In Blood 6 December 2014 124(21):3495-3495

Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.

Autor: Shangaris, Panicos, Loukogeorgakis, Stavros P., Subramaniam, Sindhu, Flouri, Christina, Jackson, Laurence H., Wang, Wei, Blundell, Michael P., Liu, Shanrun, Eaton, Simon, Bakhamis, Nahla, Ramachandra, Durrgah Latchumi, Maghsoudlou, Panayiotis, Urbani, Luca, Waddington, Simon N., Eddaoudi, Ayad, Archer, Joy, Antoniou, Michael N., Stuckey, Daniel J., Schmidt, Manfred, Thrasher, Adrian J.

Publikováno v: Scientific Reports; 12/24/2019, Vol. 9 Issue 1, p1-1, 1p

In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

Autor: Shangaris, Panicos, Loukogeorgakis, Stavros P, Subramaniam, Sindhu, Flouri, Christina, Jackson, Laurence H, Wang, Wei, Blundell, Michael P, Liu, Shanrun, Eaton, Simon, Bakhamis, Nahla, Ramachandra, Durrgah Latchumi, Maghsoudlou, Panayiotis, Urbani, Luca, Waddington, Simon N, Eddaoudi, Ayad, Archer, Joy, Antoniou, Michael N, Stuckey, Daniel J, Schmidt, Manfred, Thrasher, Adrian J, Ryan, Thomas M, De Coppi, Paolo, David, Anna L

In utero gene therapy (IUGT) to the fetal hematopoietic compartment could be used to treat congenital blood disorders such as β-thalassemia. A humanised mouse model of β-thalassemia was used, in which heterozygous animals are anaemic with splenomeg

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::11b9c768c91512e76cea429f5acd9728

In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.

Autor: Shangaris P; Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX, UK. p.shangaris@ucl.ac.uk.; UCL Institute of Child Health, UCL, London, United Kingdom. p.shangaris@ucl.ac.uk., Loukogeorgakis SP; UCL Institute of Child Health, UCL, London, United Kingdom., Subramaniam S; UCL Institute of Child Health, UCL, London, United Kingdom., Flouri C; Department of Medical and Molecular Genetics, KCL, London, United Kingdom., Jackson LH; Centre for Advanced Biomedical Imaging, UCL, London, United Kingdom., Wang W; Department of Translational Oncology, National Centre for Tumour Diseases, Heidelberg, Germany., Blundell MP; UCL Institute of Child Health, UCL, London, United Kingdom., Liu S; Biochemistry and Molecular Genetics, UAB, Birmingham, Alabama, United States., Eaton S; UCL Institute of Child Health, UCL, London, United Kingdom., Bakhamis N; Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX, UK., Ramachandra DL; UCL Institute of Child Health, UCL, London, United Kingdom., Maghsoudlou P; UCL Institute of Child Health, UCL, London, United Kingdom., Urbani L; UCL Institute of Child Health, UCL, London, United Kingdom., Waddington SN; Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX, UK.; Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa., Eddaoudi A; UCL Institute of Child Health, UCL, London, United Kingdom., Archer J; Central Diagnostic Services, Queen's Vet School Hospital, University of Cambridge, Cambridge, United Kingdom., Antoniou MN; Department of Medical and Molecular Genetics, KCL, London, United Kingdom., Stuckey DJ; Centre for Advanced Biomedical Imaging, UCL, London, United Kingdom., Schmidt M; Department of Translational Oncology, National Centre for Tumour Diseases, Heidelberg, Germany., Thrasher AJ; UCL Institute of Child Health, UCL, London, United Kingdom., Ryan TM; Biochemistry and Molecular Genetics, UAB, Birmingham, Alabama, United States., De Coppi P; UCL Institute of Child Health, UCL, London, United Kingdom., David AL; Institute for Women's Health, University College London, 86-96 Chenies Mews, London, WC1E 6HX, UK.

Publikováno v: Scientific reports [Sci Rep] 2019 Aug 12; Vol. 9 (1), pp. 11592. Date of Electronic Publication: 2019 Aug 12.