International journal of molecular sciences, 2018-09, Vol.19 (9), p.2721
2018
Details
- Autor(en) / Beteiligte
- Titel
- In Vivo Genome Editing as a Therapeutic Approach
- Ist Teil von
- International journal of molecular sciences, 2018-09, Vol.19 (9), p.2721
- Ort / Verlag
- Switzerland: MDPI AG
- Erscheinungsjahr
- 2018
- Link zum Volltext
- Quelle
- Free E-Journal (出版社公開部分のみ）
- Beschreibungen/Notizen
- Genome editing has been well established as a genome engineering tool that enables researchers to establish causal linkages between genetic mutation and biological phenotypes, providing further understanding of the genetic manifestation of many debilitating diseases. More recently, the paradigm of genome editing technologies has evolved to include the correction of mutations that cause diseases via the use of nucleases such as zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALENs), and more recently, Cas9 nuclease. With the aim of reversing disease phenotypes, which arise from somatic gene mutations, current research focuses on the clinical translatability of correcting human genetic diseases in vivo, to provide long-term therapeutic benefits and potentially circumvent the limitations of in vivo cell replacement therapy. In this review, in addition to providing an overview of the various genome editing techniques available, we have also summarized several in vivo engineering strategies that have successfully demonstrated disease correction via in vivo genome editing. The various benefits and challenges faced in applying in vivo genome editing in humans will also be discussed.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 1422-0067, 1661-6596eISSN: 1422-0067DOI: 10.3390/ijms19092721Titel-ID: cdi_doaj_primary_oai_doaj_org_article_e730b8adc30245d8b37b03a039aaf1af
- Format
- –
- Schlagworte
- Arrays, Cas9, correcting genetic mutations, CRISPR, CRISPR-Cas Systems - genetics, CRISPR-Cas Systems - physiology, Deoxyribonucleic acid, DNA, DNA repair, Drug resistance, Editing, Gene Editing - methods, Gene therapy, Genetic disorders, Genetic engineering, genome editing, Genomes, Hemophilia A - genetics, Hemophilia A - metabolism, Humans, in vivo, In vivo methods and tests, Mucopolysaccharidosis II - genetics, Mucopolysaccharidosis II - metabolism, Mutation, Mutation - genetics, Nanoparticles, Nuclease, Phenotypes, Review, TALENs, Transcription, Transcription Activator-Like Effector Nucleases - genetics, ZFN, Zinc Finger Nucleases - genetics, Zinc Finger Nucleases - metabolism, Zinc finger proteins