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Literature DB >> 21301435

Gene therapy finds its niche.

Abstract

Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens.

Entities: Disease

Mesh：

Year: 2011 PMID： 21301435 DOI： 10.1038/nbt.1769

Source DB: PubMed Journal: Nat Biotechnol ISSN： 1087-0156 Impact factor: 54.908

20 in total

Review 1. Genetic medicines: treatment strategies for hereditary disorders.

Authors: Timothy P O'Connor; Ronald G Crystal
Journal: Nat Rev Genet Date: 2006-04 Impact factor: 53.242

2. Gene therapy caught in more entanglements.

Authors: N Wade
Journal: Science Date: 1981-04-03 Impact factor: 47.728

3. 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

4. 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

5. Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Authors: Alessandro Aiuti; Federica Cattaneo; Stefania Galimberti; Ulrike Benninghoff; Barbara Cassani; Luciano Callegaro; Samantha Scaramuzza; Grazia Andolfi; Massimiliano Mirolo; Immacolata Brigida; Antonella Tabucchi; Filippo Carlucci; Martha Eibl; Memet Aker; Shimon Slavin; Hamoud Al-Mousa; Abdulaziz Al Ghonaium; Alina Ferster; Andrea Duppenthaler; Luigi Notarangelo; Uwe Wintergerst; Rebecca H Buckley; Marco Bregni; Sarah Marktel; Maria Grazia Valsecchi; Paolo Rossi; Fabio Ciceri; Roberto Miniero; Claudio Bordignon; Maria-Grazia Roncarolo
Journal: N Engl J Med Date: 2009-01-29 Impact factor: 91.245

6. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

Authors: R M Blaese; K W Culver; A D Miller; C S Carter; T Fleisher; M Clerici; G Shearer; L Chang; Y Chiang; P Tolstoshev; J J Greenblatt; S A Rosenberg; H Klein; M Berger; C A Mullen; W J Ramsey; L Muul; R A Morgan; W F Anderson
Journal: Science Date: 1995-10-20 Impact factor: 47.728

7. Gene therapy for human genetic disease?

Authors: T Friedmann; R Roblin
Journal: Science Date: 1972-03-03 Impact factor: 47.728

8. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Authors: Albert M Maguire; Katherine A High; Alberto Auricchio; J Fraser Wright; Eric A Pierce; Francesco Testa; Federico Mingozzi; Jeannette L Bennicelli; Gui-shuang Ying; Settimio Rossi; Ann Fulton; Kathleen A Marshall; Sandro Banfi; Daniel C Chung; Jessica I W Morgan; Bernd Hauck; Olga Zelenaia; Xiaosong Zhu; Leslie Raffini; Frauke Coppieters; Elfride De Baere; Kenneth S Shindler; Nicholas J Volpe; Enrico M Surace; Carmela Acerra; Arkady Lyubarsky; T Michael Redmond; Edwin Stone; Junwei Sun; Jennifer Wellman McDonnell; Bart P Leroy; Francesca Simonelli; Jean Bennett
Journal: Lancet Date: 2009-10-23 Impact factor: 79.321

9. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics.

Authors: Akin Akinc; Andreas Zumbuehl; Michael Goldberg; Elizaveta S Leshchiner; Valentina Busini; Naushad Hossain; Sergio A Bacallado; David N Nguyen; Jason Fuller; Rene Alvarez; Anna Borodovsky; Todd Borland; Rainer Constien; Antonin de Fougerolles; J Robert Dorkin; K Narayanannair Jayaprakash; Muthusamy Jayaraman; Matthias John; Victor Koteliansky; Muthiah Manoharan; Lubomir Nechev; June Qin; Timothy Racie; Denitza Raitcheva; Kallanthottathil G Rajeev; Dinah W Y Sah; Jürgen Soutschek; Ivanka Toudjarska; Hans-Peter Vornlocher; Tracy S Zimmermann; Robert Langer; Daniel G Anderson
Journal: Nat Biotechnol Date: 2008-04-27 Impact factor: 54.908

10. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.

Authors: L Naldini; U Blömer; P Gallay; D Ory; R Mulligan; F H Gage; I M Verma; D Trono
Journal: Science Date: 1996-04-12 Impact factor: 47.728

77 in total

1. Generation of human adult mesenchymal stromal/stem cells expressing defined xenogenic vascular endothelial growth factor levels by optimized transduction and flow cytometry purification.

Authors: Uta Helmrich; Anna Marsano; Ludovic Melly; Thomas Wolff; Liliane Christ; Michael Heberer; Arnaud Scherberich; Ivan Martin; Andrea Banfi
Journal: Tissue Eng Part C Methods Date: 2011-12-19 Impact factor: 3.056

2. Induction of erythropoiesis using human vascular networks genetically engineered for controlled erythropoietin release.

Authors: Ruei-Zeng Lin; Alexandra Dreyzin; Kristie Aamodt; Dan Li; Shou-Ching S Jaminet; Andrew C Dudley; Juan M Melero-Martin
Journal: Blood Date: 2011-09-21 Impact factor: 22.113

3. Gene therapy for Fanconi anemia: one step closer to the clinic.

Authors: Jakub Tolar; Pamela S Becker; D Wade Clapp; Helmut Hanenberg; Cristina Díaz de Heredia; Hans-Peter Kiem; Susana Navarro; Pankaj Qasba; Paula Rio; Manfred Schmidt; Julián Sevilla; Els Verhoeyen; Adrian J Thrasher; Juan Bueren
Journal: Hum Gene Ther Date: 2012-02 Impact factor: 5.695

Gene therapy finds its niche.

Review 1. Genetic medicines: treatment strategies for hereditary disorders.

2. Gene therapy caught in more entanglements.

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

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

5. Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

6. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

7. Gene therapy for human genetic disease?

8. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

9. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics.

10. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.

1. Generation of human adult mesenchymal stromal/stem cells expressing defined xenogenic vascular endothelial growth factor levels by optimized transduction and flow cytometry purification.

2. Induction of erythropoiesis using human vascular networks genetically engineered for controlled erythropoietin release.

3. Gene therapy for Fanconi anemia: one step closer to the clinic.

Review 4. The duality of oncomiR addiction in the maintenance and treatment of cancer.

5. TAT-HSP70 Attenuates Experimental Lung Injury.

Review 6. On methodological standards in training and transfer experiments.

7. Gene therapy, early promises, subsequent problems, and recent breakthroughs.

Review 8. Protein engineering: a new frontier for biological therapeutics.

Review 9. Barriers to inhaled gene therapy of obstructive lung diseases: A review.

10. Non-viral gene-activated matrices: next generation constructs for bone repair.