Literature DB >> 21959273

Gene therapies advance towards finish line.

Asher Mullard.   

Abstract

Mesh：

Year:  2011        PMID： 21959273     DOI： 10.1038/nrd3572

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN： 1474-1776            Impact factor:   84.694


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  9 in total

1.  Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia.

Authors:  David L Porter; Bruce L Levine; Michael Kalos; Adam Bagg; Carl H June
Journal:  N Engl J Med       Date:  2011-08-10       Impact factor: 91.245

Review 2.  State-of-the-art gene-based therapies: the road ahead.

Authors:  Mark A Kay
Journal:  Nat Rev Genet       Date:  2011-04-06       Impact factor: 53.242

3.  Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.

Authors:  H Bobby Gaspar; Samantha Cooray; Kimberly C Gilmour; Kathryn L Parsley; Fang Zhang; Stuart Adams; Emma Bjorkegren; Jinhua Bayford; Lucinda Brown; E Graham Davies; Paul Veys; Lynette Fairbanks; Victoria Bordon; Theoni Petropoulou; Theoni Petropolou; Christine Kinnon; Adrian J Thrasher
Journal:  Sci Transl Med       Date:  2011-08-24       Impact factor: 17.956

4.  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

5.  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

6.  Stem-cell gene therapy for the Wiskott-Aldrich syndrome.

Authors:  Kaan Boztug; Manfred Schmidt; Adrian Schwarzer; Pinaki P Banerjee; Inés Avedillo Díez; Ricardo A Dewey; Marie Böhm; Ali Nowrouzi; Claudia R Ball; Hanno Glimm; Sonja Naundorf; Klaus Kühlcke; Rainer Blasczyk; Irina Kondratenko; László Maródi; Jordan S Orange; Christof von Kalle; Christoph Klein
Journal:  N Engl J Med       Date:  2010-11-11       Impact factor: 91.245

7.  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

8.  Safety and efficacy of gene transfer for Leber's congenital amaurosis.

Authors:  Albert M Maguire; Francesca Simonelli; Eric A Pierce; Edward N Pugh; Federico Mingozzi; Jeannette Bennicelli; Sandro Banfi; Kathleen A Marshall; Francesco Testa; Enrico M Surace; Settimio Rossi; Arkady Lyubarsky; Valder R Arruda; Barbara Konkle; Edwin Stone; Junwei Sun; Jonathan Jacobs; Lou Dell'Osso; Richard Hertle; Jian-xing Ma; T Michael Redmond; Xiaosong Zhu; Bernd Hauck; Olga Zelenaia; Kenneth S Shindler; Maureen G Maguire; J Fraser Wright; Nicholas J Volpe; Jennifer Wellman McDonnell; Alberto Auricchio; Katherine A High; Jean Bennett
Journal:  N Engl J Med       Date:  2008-04-27       Impact factor: 91.245

9.  Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.

Authors:  H Bobby Gaspar; Samantha Cooray; Kimberly C Gilmour; Kathryn L Parsley; Stuart Adams; Steven J Howe; Abdulaziz Al Ghonaium; Jinhua Bayford; Lucinda Brown; E Graham Davies; Christine Kinnon; Adrian J Thrasher
Journal:  Sci Transl Med       Date:  2011-08-24       Impact factor: 17.956
  9 in total
  3 in total

1.  Glybera and the future of gene therapy in the European Union.

Authors:  Norman Miller
Journal:  Nat Rev Drug Discov       Date:  2012-05       Impact factor: 84.694

Review 2.  Drug discovery and development for rare genetic disorders.

Authors:  Wei Sun; Wei Zheng; Anton Simeonov
Journal:  Am J Med Genet A       Date:  2017-07-21       Impact factor: 2.802

3.  Next-generation muscle-directed gene therapy by in silico vector design.

Authors:  S Sarcar; W Tulalamba; M Y Rincon; J Tipanee; H Q Pham; H Evens; D Boon; E Samara-Kuko; M Keyaerts; M Loperfido; E Berardi; S Jarmin; P In't Veld; G Dickson; T Lahoutte; M Sampaolesi; P De Bleser; T VandenDriessche; M K Chuah
Journal:  Nat Commun       Date:  2019-01-30       Impact factor: 14.919
  3 in total

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