Literature DB >> 12897195

Targeted genetic repair: an emerging approach to genetic therapy.

Bruce A Sullenger1.   

Abstract

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Year:  2003        PMID： 12897195      PMCID： PMC166306          DOI： 10.1172/JCI19419

Source DB:  PubMed          Journal:  J Clin Invest        ISSN： 0021-9738            Impact factor:   14.808


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  21 in total

1.  Repairing the sickle cell mutation. I. Specific covalent binding of a photoreactive third strand to the mutated base pair.

Authors:  S Broitman; O Amosova; N G Dolinnaya; J R Fresco
Journal:  J Biol Chem       Date:  1999-07-30       Impact factor: 5.157

2.  Functional repair of a mutant chloride channel using a trans-splicing ribozyme.

Authors:  Christopher S Rogers; Carlos G Vanoye; Bruce A Sullenger; Alfred L George
Journal:  J Clin Invest       Date:  2002-12       Impact factor: 14.808

3.  A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.

Authors:  Salima Hacein-Bey-Abina; Christof von Kalle; Manfred Schmidt; Françoise Le Deist; Nicolas Wulffraat; Elisabeth McIntyre; Isabelle Radford; Jean-Luc Villeval; Christopher C Fraser; Marina Cavazzana-Calvo; Alain Fischer
Journal:  N Engl J Med       Date:  2003-01-16       Impact factor: 91.245

Review 4.  Ribozyme-mediated revision of RNA and DNA.

Authors:  Meredith B Long; J P Jones; Bruce A Sullenger; Jonghoe Byun
Journal:  J Clin Invest       Date:  2003-08       Impact factor: 14.808

5.  Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing.

Authors:  Xiaoming Liu; Qinshi Jiang; S Gary Mansfield; M Puttaraju; Yulong Zhang; Weihong Zhou; Jonathan A Cohn; Mariano A Garcia-Blanco; Lloyd G Mitchell; John F Engelhardt
Journal:  Nat Biotechnol       Date:  2002-01       Impact factor: 54.908

6.  Application of SFHR to gene therapy of monogenic disorders.

Authors:  K K Goncz; N L Prokopishyn; B L Chow; B R Davis; D C Gruenert
Journal:  Gene Ther       Date:  2002-06       Impact factor: 5.250

7.  Induction of wild-type p53 activity in human cancer cells by ribozymes that repair mutant p53 transcripts.

Authors:  T Watanabe; B A Sullenger
Journal:  Proc Natl Acad Sci U S A       Date:  2000-07-18       Impact factor: 11.205

8.  Expression of DeltaF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR.

Authors:  K K Goncz; A Colosimo; B Dallapiccola; L Gagné; K Hong; G Novelli; D Papahadjopoulos; T Sawa; H Schreier; J Wiener-Kronish; Z Xu; D C Gruenert
Journal:  Gene Ther       Date:  2001-06       Impact factor: 5.250

9.  Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.

Authors:  S G Mansfield; J Kole; M Puttaraju; C C Yang; M A Garcia-Blanco; J A Cohn; L G Mitchell
Journal:  Gene Ther       Date:  2000-11       Impact factor: 5.250

10.  Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder.

Authors:  Thipparat Suwanmanee; Halina Sierakowska; Suthat Fucharoen; Ryszard Kole
Journal:  Mol Ther       Date:  2002-12       Impact factor: 11.454
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  4 in total

1.  Increased efficiency of oligonucleotide-mediated gene repair through slowing replication fork progression.

Authors:  Xue-Song Wu; Li Xin; Wen-Xuan Yin; Xi-Ying Shang; Lu Lu; Rory M Watt; Kathryn S E Cheah; Jian-Dong Huang; De-Pei Liu; Chih-Chuan Liang
Journal:  Proc Natl Acad Sci U S A       Date:  2005-02-04       Impact factor: 11.205

Review 2.  Cftr gene targeting in mouse embryonic stem cells mediated by Small Fragment Homologous Replacement (SFHR).

Authors:  Federica Sangiuolo; Maria Lucia Scaldaferri; Antonio Filareto; Paola Spitalieri; Lorenzo Guerra; Maria Favia; Rosa Caroppo; Ruggiero Mango; Emanuela Bruscia; Dieter C Gruenert; Valeria Casavola; Massimo De Felici; Giuseppe Novelli
Journal:  Front Biosci       Date:  2008-01-01

3.  Effect of Steric Constraint at the γ-Backbone Position on the Conformations and Hybridization Properties of PNAs.

Authors:  Matthew J Crawford; Srinivas Rapireddy; Raman Bahal; Iulia Sacui; Danith H Ly
Journal:  J Nucleic Acids       Date:  2011-05-25

4.  The involvement of replication in single stranded oligonucleotide-mediated gene repair.

Authors:  Michael S Y Huen; Xin-tian Li; Lin-Yu Lu; Rory M Watt; De-Pei Liu; Jian-Dong Huang
Journal:  Nucleic Acids Res       Date:  2006-11-06       Impact factor: 16.971
  4 in total

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