Gene therapy progress and prospects: targeted gene repair
- 1 April 2005
- journal article
- review article
- Published by Springer Nature in Gene Therapy
- Vol. 12 (8) , 639-646
- https://doi.org/10.1038/sj.gt.3302511
Abstract
The capacity to correct a mutant gene within the context of the chromosome holds great promise as a therapy for inherited disorders but fulfilling this promise has proven to be challenging. However, steady progress is being made and the development of gene repair as a viable and robust approach is underway. Here, we present some of the recent advances that are helping to shape our thinking about the feasibility and the limitations of this technique. For the most part, these advances center on understanding the regulation of the reaction and validating its application in animal models.Keywords
This publication has 50 references indexed in Scilit:
- Strand bias in oligonucleotide-mediated dystrophin gene editingHuman Molecular Genetics, 2004
- Enhanced oligonucleotide-directed gene targeting in mammalian cells following treatment with DNA damaging agentsExperimental Cell Research, 2004
- DNA replication and transcription direct a DNA strand bias in the process of targeted gene repair in mammalian cellsJournal of Cell Science, 2004
- Nuclease Activity of Saccharomyces cerevisiae Mre11 Functions in Targeted Nucleotide AlterationApplied and Environmental Microbiology, 2003
- Branched oligonucleotides induce in vivo gene conversion of a mutated EGFP reporterGene Therapy, 2003
- The development and regulation of gene repairNature Reviews Genetics, 2003
- Progress and problems with the use of viral vectors for gene therapyNature Reviews Genetics, 2003
- Site-specific genomic integration produces therapeutic Factor IX levels in miceNature Biotechnology, 2002
- Functional correction of episomal mutations withshort DNA fragments and RNA–DNA oligonucleotidesThe Journal of Gene Medicine, 2002
- Targeted beta-globin gene conversion in human hematopoietic CD34+ and Lin−CD38− cellsGene Therapy, 2002