International journal of molecular sciences, 2020-05, Vol.21 (11), p.3903
2020
Details
- Autor(en) / Beteiligte
- Titel
- Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing
- Ist Teil von
- International journal of molecular sciences, 2020-05, Vol.21 (11), p.3903
- Ort / Verlag
- Switzerland: MDPI AG
- Erscheinungsjahr
- 2020
- Link zum Volltext
- Quelle
- Free E-Journal (出版社公開部分のみ）
- Beschreibungen/Notizen
- Since the early days of its conceptualization and application, human gene transfer held the promise of a permanent solution to genetic diseases including cystic fibrosis (CF). This field went through alternated periods of enthusiasm and distrust. The development of refined technologies allowing site specific modification with programmable nucleases highly revived the gene therapy field. CRISPR nucleases and derived technologies tremendously facilitate genome manipulation offering diversified strategies to reverse mutations. Here we discuss the advancement of gene therapy, from therapeutic nucleic acids to genome editing techniques, designed to reverse genetic defects in CF. We provide a roadmap through technologies and strategies tailored to correct different types of mutations in the cystic fibrosis transmembrane regulator ( gene, and their applications for the development of experimental models valuable for the advancement of CF therapies.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 1422-0067, 1661-6596eISSN: 1422-0067DOI: 10.3390/ijms21113903Titel-ID: cdi_doaj_primary_oai_doaj_org_article_fc238cd8ff1d4549b9253f3f83a699e4
- Format
- –
- Schlagworte
- Animals, Blood diseases, Clinical trials, CRISPR, CRISPR-Cas, CRISPR-Cas Systems, Cystic fibrosis, Cystic Fibrosis - therapy, Cystic fibrosis transmembrane conductance regulator, Cystic Fibrosis Transmembrane Conductance Regulator - genetics, Deoxyribonucleic acid, Disease, Disease Models, Animal, DNA, Editing, Enzymes, Gene Editing, Gene therapy, Genetic disorders, Genetic engineering, Genetic Therapy - methods, Genetic Therapy - trends, genome editing, Genome, Human, Genomes, Humans, Mice, Mortality, Mutagenesis, Mutation, Nuclease, Nucleic acids, Oligonucleotides, Antisense, Phenotype, programmable nucleases, Review, Vectors (Biology)