Literature DB >> 16763910

Gene therapy of metabolic diseases.

Alain Fischer1, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo.   

Abstract

This review surveys the potential application of gene therapy to metabolic diseases. Proof of principle has been achieved in the treatment of two inherited immunodeficiency conditions. A significant safety issue has also been observed. Several strategies are being experimentally tested for a number of metabolic diseases (more than 20) and may provide a rationale for the future development of clinical trials.

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Year:  2006        PMID： 16763910     DOI： 10.1007/s10545-006-0270-7

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN： 0141-8955            Impact factor:   4.982


  23 in total

Review 1.  Harnessing HIV for therapy, basic research and biotechnology.

Authors:  Maciej Wiznerowicz; Didier Trono
Journal:  Trends Biotechnol       Date:  2005-01       Impact factor: 19.536

Review 2.  Gene targeting using zinc finger nucleases.

Authors:  Matthew H Porteus; Dana Carroll
Journal:  Nat Biotechnol       Date:  2005-08       Impact factor: 54.908

3.  Highly efficient endogenous human gene correction using designed zinc-finger nucleases.

Authors:  Fyodor D Urnov; Jeffrey C Miller; Ya-Li Lee; Christian M Beausejour; Jeremy M Rock; Sheldon Augustus; Andrew C Jamieson; Matthew H Porteus; Philip D Gregory; Michael C Holmes
Journal:  Nature       Date:  2005-04-03       Impact factor: 49.962

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.  Site-specific genomic integration produces therapeutic Factor IX levels in mice.

Authors:  Eric C Olivares; Roger P Hollis; Thomas W Chalberg; Leonard Meuse; Mark A Kay; Michele P Calos
Journal:  Nat Biotechnol       Date:  2002-10-15       Impact factor: 54.908

6.  Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice.

Authors:  Sonia Benhamida; Françoise Pflumio; Anne Dubart-Kupperschmitt; Jing Chao Zhao-Emonet; Marina Cavazzana-Calvo; Francis Rocchiccioli; Serge Fichelson; Patrick Aubourg; Pierre Charneau; Nathalie Cartier
Journal:  Mol Ther       Date:  2003-03       Impact factor: 11.454

7.  Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial.

Authors:  Manfred Schmidt; Salima Hacein-Bey-Abina; Manuela Wissler; Frederique Carlier; Annick Lim; Claudia Prinz; Hanno Glimm; Isabelle Andre-Schmutz; Christophe Hue; Alexandrine Garrigue; Francoise Le Deist; Chantal Lagresle; Alain Fischer; Marina Cavazzana-Calvo; Christof von Kalle
Journal:  Blood       Date:  2004-12-07       Impact factor: 22.113

8.  Successful gene therapy of the Gunn rat by in vivo neonatal hepatic gene transfer using murine oncoretroviral vectors.

Authors:  Marta Bellodi-Privato; Dominique Aubert; Virginie Pichard; Anne Myara; François Trivin; Nicolas Ferry
Journal:  Hepatology       Date:  2005-08       Impact factor: 17.425

9.  Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency.

Authors:  Frank Yates; Michèle Malassis-Séris; Daniel Stockholm; Cécile Bouneaud; Frédérique Larousserie; Patricia Noguiez-Hellin; Olivier Danos; Donald B Kohn; Alain Fischer; Jean-Pierre de Villartay; Marina Cavazzana-Calvo
Journal:  Blood       Date:  2002-08-22       Impact factor: 22.113

10.  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
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  1 in total

1.  Processing of triplex-directed psoralen DNA interstrand crosslinks by recombination mechanisms.

Authors:  Yaobin Liu; Rodney S Nairn; Karen M Vasquez
Journal:  Nucleic Acids Res       Date:  2008-07-15       Impact factor: 16.971
  1 in total

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