PubMed · 14918532
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J KOTAS. 1952-01-15. [Reticulosarcoma].. https://pubmed.ncbi.nlm.nih.gov/14918532/
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Many common neurological disorders, such as Parkinson's disease, stroke and multiple sclerosis, are caused by a loss of neurons and glial cells. In recent years, neurons and glia have been generated successfully from stem cells in culture, fueling efforts to develop stem-cell-based transplantation therapies for human patients. More recently, efforts have been extended to stimulating the formation and preventing the death of neurons and glial cells produced by endogenous stem cells within the adult central nervous system. The next step is to translate these exciting advances from the laboratory into clinically useful therapies.
For decades, transplantation of haematopoietic stem cells--either unmodified, or genetically modified to correct genetic disorders--has been used to treat disorders of the blood and immune systems. The present challenge is to reduce the risk of such transplants and increase the number of patients who can safely access this treatment. In developing countries, such 'one-shot' treatments are highly desirable because chronic treatments are difficult to sustain. To make these therapies more accessible and effective it will be important to improve clinical protocols and gene-delivery vectors, and to gain a deeper understanding of stem cells.
Since the discovery of the CF gene over a decade ago, several groups worldwide have explored the potential of gene therapy, the insertion of a normal copy of the gene into the respiratory epithelium. Both viral and synthetic gene transfer agents have been designed to this end, although problems with repeat application have occurred with the former have occurred. Here, we will briefly review success in clinical trials to date. Cell therapy, replacement of the CF cell with a healthy, normal cell, is at a much earlier stage of development, studies mainly being preclinical or observational. Some evidence of plasticity of, for example, bone marrow cells, has been demonstrated, although for the airway, levels of correction appear to be extremely low and a degree of damage seems to be a prerequisite. Whether this approach will be able to achieve clinical success remains to be seen.