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Literature DB >> 20844523

Gene therapy: Targeting β-thalassaemia.

Derek A Persons.

Abstract

Entities: Disease

Mesh：

Substances：

Year: 2010 PMID： 20844523 DOI： 10.1038/467277a

Source DB: PubMed Journal: Nature ISSN： 0028-0836 Impact factor: 49.962

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10 in total

1. Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH).

Authors: Norimitsu Inoue; Tomohisa Izui-Sarumaru; Yoshiko Murakami; Yuichi Endo; Jun-Ichi Nishimura; Ken Kurokawa; Maki Kuwayama; Hiroaki Shime; Takashi Machii; Yuzuru Kanakura; Gabrielle Meyers; Carl Wittwer; Zhong Chen; William Babcock; Debra Frei-Lahr; Charles J Parker; Taroh Kinoshita
Journal: Blood Date: 2006-08-29 Impact factor: 22.113

2. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Authors: M Cavazzana-Calvo; S Hacein-Bey; G de Saint Basile; F Gross; E Yvon; P Nusbaum; F Selz; C Hue; S Certain; J L Casanova; P Bousso; F L Deist; A Fischer
Journal: Science Date: 2000-04-28 Impact factor: 47.728

3. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

Authors: Marina Cavazzana-Calvo; Emmanuel Payen; Olivier Negre; Gary Wang; Kathleen Hehir; Floriane Fusil; Julian Down; Maria Denaro; Troy Brady; Karen Westerman; Resy Cavallesco; Beatrix Gillet-Legrand; Laure Caccavelli; Riccardo Sgarra; Leila Maouche-Chrétien; Françoise Bernaudin; Robert Girot; Ronald Dorazio; Geert-Jan Mulder; Axel Polack; Arthur Bank; Jean Soulier; Jérôme Larghero; Nabil Kabbara; Bruno Dalle; Bernard Gourmel; Gérard Socie; Stany Chrétien; Nathalie Cartier; Patrick Aubourg; Alain Fischer; Kenneth Cornetta; Frédéric Galacteros; Yves Beuzard; Eliane Gluckman; Frederick Bushman; Salima Hacein-Bey-Abina; Philippe Leboulch
Journal: Nature Date: 2010-09-16 Impact factor: 49.962

4. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

Authors: Marion G Ott; Manfred Schmidt; Kerstin Schwarzwaelder; Stefan Stein; Ulrich Siler; Ulrike Koehl; Hanno Glimm; Klaus Kühlcke; Andrea Schilz; Hana Kunkel; Sonja Naundorf; Andrea Brinkmann; Annette Deichmann; Marlene Fischer; Claudia Ball; Ingo Pilz; Cynthia Dunbar; Yang Du; Nancy A Jenkins; Neal G Copeland; Ursula Lüthi; Moustapha Hassan; Adrian J Thrasher; Dieter Hoelzer; Christof von Kalle; Reinhard Seger; Manuel Grez
Journal: Nat Med Date: 2006-04-02 Impact factor: 53.440

5. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

Authors: S Hacein-Bey-Abina; C Von Kalle; M Schmidt; M P McCormack; N Wulffraat; P Leboulch; A Lim; C S Osborne; R Pawliuk; E Morillon; R Sorensen; A Forster; P Fraser; J I Cohen; G de Saint Basile; I Alexander; U Wintergerst; T Frebourg; A Aurias; D Stoppa-Lyonnet; S Romana; I Radford-Weiss; F Gross; F Valensi; E Delabesse; E Macintyre; F Sigaux; J Soulier; L E Leiva; M Wissler; C Prinz; T H Rabbitts; F Le Deist; A Fischer; M Cavazzana-Calvo
Journal: Science Date: 2003-10-17 Impact factor: 47.728

Review 6. Roles of HMGA proteins in cancer.

Authors: Alfredo Fusco; Monica Fedele
Journal: Nat Rev Cancer Date: 2007-12 Impact factor: 60.716

7. Prospects for human gene therapy.

Authors: W F Anderson
Journal: Science Date: 1984-10-26 Impact factor: 47.728

8. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.

Authors: Nathalie Cartier; Salima Hacein-Bey-Abina; Cynthia C Bartholomae; Gabor Veres; Manfred Schmidt; Ina Kutschera; Michel Vidaud; Ulrich Abel; Liliane Dal-Cortivo; Laure Caccavelli; Nizar Mahlaoui; Véronique Kiermer; Denice Mittelstaedt; Céline Bellesme; Najiba Lahlou; François Lefrère; Stéphane Blanche; Muriel Audit; Emmanuel Payen; Philippe Leboulch; Bruno l'Homme; Pierre Bougnères; Christof Von Kalle; Alain Fischer; Marina Cavazzana-Calvo; Patrick Aubourg
Journal: Science Date: 2009-11-06 Impact factor: 47.728

9. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.

Authors: Alessandro Aiuti; Shimon Slavin; Memet Aker; Francesca Ficara; Sara Deola; Alessandra Mortellaro; Shoshana Morecki; Grazia Andolfi; Antonella Tabucchi; Filippo Carlucci; Enrico Marinello; Federica Cattaneo; Sergio Vai; Paolo Servida; Roberto Miniero; Maria Grazia Roncarolo; Claudio Bordignon
Journal: Science Date: 2002-06-28 Impact factor: 47.728

10. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration.

Authors: Eugenio Montini; Daniela Cesana; Manfred Schmidt; Francesca Sanvito; Maurilio Ponzoni; Cynthia Bartholomae; Lucia Sergi Sergi; Fabrizio Benedicenti; Alessandro Ambrosi; Clelia Di Serio; Claudio Doglioni; Christof von Kalle; Luigi Naldini
Journal: Nat Biotechnol Date: 2006-05-28 Impact factor: 54.908

10 in total

8 in total

Gene therapy: Targeting β-thalassaemia.

1. Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH).

2. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

3. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

4. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

5. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

Review 6. Roles of HMGA proteins in cancer.

7. Prospects for human gene therapy.

8. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.

9. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.

10. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration.

Review 1. Development of gene therapy for thalassemia.

2. Efficient gene delivery of primary human cells using peptide linked polyethylenimine polymer hybrid.

Review 3. Development and clinical translation of ex vivo gene therapy.

4. In vivo activation of the human δ-globin gene: the therapeutic potential in β-thalassemic mice.

Review 5. α-Globin as a molecular target in the treatment of β-thalassemia.

6. miR-451 Up-regulation, Induce Erythroid Differentiation of CD133+cells Independent of Cytokine Cocktails.

7. Gene Therapy: A Possible Alternative to CFTR Modulators?

Review 8. Novel genetic therapeutic approaches for modulating the severity of β-thalassemia (Review).