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Gene, Stem Cell, and Future Therapies for Orphan Diseases
Author(s) -
Phillips M Ian
Publication year - 2012
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1038/clpt.2012.82
Subject(s) - stem cell , genetic enhancement , induced pluripotent stem cell , zinc finger nuclease , stem cell therapy , cell therapy , medicine , biology , genome editing , cancer research , transcription activator like effector nuclease , bioinformatics , embryonic stem cell , gene , genetics , crispr
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