Literature DB >> 12952907

Targeted gene repair -- in the arena.

Eric B Kmiec1.   

Abstract

The development of targeted gene repair is under way and, despite some setbacks, shows promise as an alternative form of gene therapy. This approach uses synthetic DNA molecules to activate and direct the cell's inherent DNA repair systems to correct inborn errors. The progress of this technique and its therapeutic potential are discussed in relation to the treatment of genetic diseases.

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Year:  2003        PMID： 12952907      PMCID： PMC182220          DOI： 10.1172/JCI19777

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


  51 in total

1.  High efficiency mutagenesis, repair, and engineering of chromosomal DNA using single-stranded oligonucleotides.

Authors:  H M Ellis; D Yu; T DiTizio; D L Court
Journal:  Proc Natl Acad Sci U S A       Date:  2001-05-29       Impact factor: 11.205

2.  Correction of a human beta S-globin gene by gene targeting.

Authors:  E G Shesely; H S Kim; W R Shehee; T Papayannopoulou; O Smithies; B W Popovich
Journal:  Proc Natl Acad Sci U S A       Date:  1991-05-15       Impact factor: 11.205

3.  Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides and modified single-stranded vectors.

Authors:  H Parekh-Olmedo; K Czymmek; E B Kmiec
Journal:  Sci STKE       Date:  2001-03-13

4.  Correction of chromosomal point mutations in human cells with bifunctional oligonucleotides.

Authors:  K W Culver; W T Hsieh; Y Huyen; V Chen; J Liu; Y Khripine; A Khorlin
Journal:  Nat Biotechnol       Date:  1999-10       Impact factor: 54.908

5.  In vitro and in vivo nucleotide exchange directed by chimeric RNA/DNA oligonucleotides in Saccharomyces cerevisae.

Authors:  M C Rice; M Bruner; K Czymmek; E B Kmiec
Journal:  Mol Microbiol       Date:  2001-05       Impact factor: 3.501

6.  Targeted correction of the point mutations of beta-thalassemia and targeted mutagenesis of the nucleotide associated with HPFH by RNA/DNA oligonucleotides: potential for beta-thalassemia gene therapy.

Authors:  Z H Li; D P Liu; W X Yin; Z C Guo; C C Liang
Journal:  Blood Cells Mol Dis       Date:  2001 Mar-Apr       Impact factor: 3.039

7.  In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides.

Authors:  L Liu; M C Rice; E B Kmiec
Journal:  Nucleic Acids Res       Date:  2001-10-15       Impact factor: 16.971

8.  Parameters affecting the frequencies of transformation and co-transformation with synthetic oligonucleotides in yeast.

Authors:  T Yamamoto; R P Moerschell; L P Wakem; D Ferguson; F Sherman
Journal:  Yeast       Date:  1992-11       Impact factor: 3.239

9.  Strand-specificity in the transformation of yeast with synthetic oligonucleotides.

Authors:  T Yamamoto; R P Moerschell; L P Wakem; S Komar-Panicucci; F Sherman
Journal:  Genetics       Date:  1992-08       Impact factor: 4.562

Review 10.  Gene targeting approaches to complex genetic diseases: atherosclerosis and essential hypertension.

Authors:  O Smithies; N Maeda
Journal:  Proc Natl Acad Sci U S A       Date:  1995-06-06       Impact factor: 11.205
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  8 in total

Review 1.  Targeted genetic repair: an emerging approach to genetic therapy.

Authors:  Bruce A Sullenger
Journal:  J Clin Invest       Date:  2003-08       Impact factor: 14.808

2.  Correction of chromosomal mutation and random integration in embryonic stem cells with helper-dependent adenoviral vectors.

Authors:  Fumi Ohbayashi; Michael A Balamotis; Atsuhiro Kishimoto; Emi Aizawa; Arturo Diaz; Paul Hasty; Frank L Graham; C Thomas Caskey; Kohnosuke Mitani
Journal:  Proc Natl Acad Sci U S A       Date:  2005-09-07       Impact factor: 11.205

3.  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

4.  RecA-independent single-stranded DNA oligonucleotide-mediated mutagenesis.

Authors:  Kenan C Murphy; Martin G Marinus
Journal:  F1000 Biol Rep       Date:  2010-07-22

5.  Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation.

Authors:  Dan Wang; Charu Shukla; Xiaoli Liu; Trenton R Schoeb; Lorne A Clarke; David M Bedwell; Kim M Keeling
Journal:  Mol Genet Metab       Date:  2010-01       Impact factor: 4.797

6.  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

7.  Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.

Authors:  Sandra P Melo; Leszek Lisowski; Elizaveta Bashkirova; Hanson H Zhen; Kirk Chu; Douglas R Keene; M Peter Marinkovich; Mark A Kay; Anthony E Oro
Journal:  Mol Ther       Date:  2014-01-06       Impact factor: 11.454

8.  Prevention of Transcriptional γ-globin Gene Silencing by Inducing The Hereditary Persistence of Fetal Hemoglobin Point Mutation Using Chimeraplast-Mediated Gene Targeting.

Authors:  Reza Ranjbaran; Mahin Nikogoftar Zarif; Sedigheh Sharifzadeh; Habibollah Golafshan; Ali Akbar Pourfathollah
Journal:  Cell J       Date:  2018-05-15       Impact factor: 2.479
  8 in total

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