Gene, Stem Cell, and Future Therapies for Orphan Diseases
| Autor: | M. Ian Phillips |
|---|---|
| Rok vydání: | 2012 |
| Předmět: |
Pharmacology
Genetics Clinical Trials as Topic DNA Repair DNA repair Effector Genetic enhancement medicine.medical_treatment Genetic Therapy Stem-cell therapy Biology Paracrine signalling Rare Diseases Gene Targeting Cancer research medicine Humans Pharmacology (medical) Stem cell Induced pluripotent stem cell Gene Stem Cell Transplantation |
| Zdroj: | Clinical Pharmacology & Therapeutics. 92:182-192 |
| ISSN: | 1532-6535 0009-9236 |
| Popis: | There are an estimated 7,000 “orphan diseases,” but treatments are currently available for only about 5% of them. Recent progress in the advanced platforms of gene therapy, stem cell therapy, gene modification, and gene correction offers possibilities for new therapies and cures for rare diseases. Many rare diseases are genetic in origin, and gene therapy is being successfully applied to treat them. Human stem cell therapy, apart from bone marrow transplants, is still experimental. Genetic modification of stem cells can make stem cell–based products more effective. Autologous induced pluripotent stem (iPS) cells, when combined with new classes of artificial nucleases, have great potential in the ex vivo repair of specific mutated DNA sequences (zinc-finger proteins and transactivator-like effector nucleases). Patient-specific iPS cells can be corrected and transplanted back into the patient. Stem cells secrete paracrine factors that could become new therapeutic tools in the treatment of orphan diseases. Gene therapy and stem cell therapy with DNA repair are promising approaches to the treatment of rare, intractable diseases. Clinical Pharmacology & Therapeutics (2012); 92 2, 182–192. doi:10.1038/clpt.2012.82 |
| Databáze: | OpenAIRE |
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