Literature DB >> 20118988

Gene therapy: back on track?

Fulvio Mavilio1.   

Abstract

Mesh：

Year:  2010        PMID： 20118988      PMCID： PMC2828753          DOI： 10.1038/embor.2009.284

Source DB:  PubMed          Journal:  EMBO Rep        ISSN： 1469-221X            Impact factor:   8.807


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  7 in total

1.  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

2.  Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

Authors:  Salima Hacein-Bey-Abina; Alexandrine Garrigue; Gary P Wang; Jean Soulier; Annick Lim; Estelle Morillon; Emmanuelle Clappier; Laure Caccavelli; Eric Delabesse; Kheira Beldjord; Vahid Asnafi; Elizabeth MacIntyre; Liliane Dal Cortivo; Isabelle Radford; Nicole Brousse; François Sigaux; Despina Moshous; Julia Hauer; Arndt Borkhardt; Bernd H Belohradsky; Uwe Wintergerst; Maria C Velez; Lily Leiva; Ricardo Sorensen; Nicolas Wulffraat; Stéphane Blanche; Frederic D Bushman; Alain Fischer; Marina Cavazzana-Calvo
Journal:  J Clin Invest       Date:  2008-09       Impact factor: 14.808

3.  Effect of gene therapy on visual function in Leber's congenital amaurosis.

Authors:  James W B Bainbridge; Alexander J Smith; Susie S Barker; Scott Robbie; Robert Henderson; Kamaljit Balaggan; Ananth Viswanathan; Graham E Holder; Andrew Stockman; Nick Tyler; Simon Petersen-Jones; Shomi S Bhattacharya; Adrian J Thrasher; Fred W Fitzke; Barrie J Carter; Gary S Rubin; Anthony T Moore; Robin R Ali
Journal:  N Engl J Med       Date:  2008-04-27       Impact factor: 91.245

4.  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

5.  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

6.  Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study.

Authors:  Fabio Ciceri; Chiara Bonini; Maria Teresa Lupo Stanghellini; Attilio Bondanza; Catia Traversari; Monica Salomoni; Lucia Turchetto; Scialini Colombi; Massimo Bernardi; Jacopo Peccatori; Alessandra Pescarollo; Paolo Servida; Zulma Magnani; Serena K Perna; Veronica Valtolina; Fulvio Crippa; Luciano Callegaro; Elena Spoldi; Roberto Crocchiolo; Katharina Fleischhauer; Maurilio Ponzoni; Luca Vago; Silvano Rossini; Armando Santoro; Elisabetta Todisco; Jane Apperley; Eduardo Olavarria; Shimon Slavin; Eva M Weissinger; Arnold Ganser; Michael Stadler; Evangelia Yannaki; Athanasios Fassas; Achilles Anagnostopoulos; Marco Bregni; Corrado Gallo Stampino; Paolo Bruzzi; Claudio Bordignon
Journal:  Lancet Oncol       Date:  2009-04-01       Impact factor: 41.316

Review 7.  A look to future directions in gene therapy research for monogenic diseases.

Authors:  Matthew H Porteus; Jon P Connelly; Shondra M Pruett
Journal:  PLoS Genet       Date:  2006-09-29       Impact factor: 5.917
  7 in total
  2 in total

1.  In vivo gene delivery with L-tyrosine polyphosphate nanoparticles.

Authors:  Andrew J Ditto; John J Reho; Kush N Shah; Justin A Smolen; James H Holda; Rolando J Ramirez; Yang H Yun
Journal:  Mol Pharm       Date:  2013-04-08       Impact factor: 4.939

2.  A single epidermal stem cell strategy for safe ex vivo gene therapy.

Authors:  Stéphanie Droz-Georget Lathion; Ariane Rochat; Graham Knott; Alessandra Recchia; Danielle Martinet; Sara Benmohammed; Nicolas Grasset; Andrea Zaffalon; Nathalie Besuchet Schmutz; Emmanuelle Savioz-Dayer; Jacques Samuel Beckmann; Jacques Rougemont; Fulvio Mavilio; Yann Barrandon
Journal:  EMBO Mol Med       Date:  2015-04       Impact factor: 12.137
  2 in total

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