The first clinical trials using retroviral vectors demonstrated the feasibility and social and regulatory acceptance of gene transfer into human subject. The initial trials have greatly expanded interest in gene therapy and led to a more focused analysis of the potential clinical indications for gene therapy.
To date, the number of patients treated is still too small to fully assess the safety or efficacy of either retroviral vectors or any specific gene therapy.
Gene replacement is now commonly performed in research laboratories studying inborn errors of metabolism,
and more than 100 diseases have been “curred” in experiments performed in cells cultured from affected patients. The list of potential diseases for gene therapy now encompasses virtually any inborn error of metabolism for which the gene has been cloned,
and more than 100 diseases have been “curred” in experiments performed in cells cultured from affected patients. The list of potential diseases for gene therapy now encompasses virtually any inborn error of metabolism for which the gene has been cloned,
the necessary level of expression of the therapeutic gene is achievable, and the target cell is accessible. Gene derived cells has been achieved, and a variety of metabolic diseases which affect these organs may be considered realistic candidates for gene therapy. At the present time, however, the fraction of cells in any organ that can be effectively targeted by vector delivery is limited, and the levels of expression that have been achieved in vivo are relatively low.
With the increased understanding of, both, the potential and limitations of gene therapy, several diseases which were initially considered attractive candidates for gene therapy are now considered less suitable. For example, current methods may not produce sufficiently high expression of hemoglobin to treat thalasemia, and the diffeculy of vector delivery to the central nervous system make gene therapy for Lesch-Nyhan disease more distant, At the some time, there isi a growing realization that gene therapy may be relatively safe and that such therapy does not need to be restricted to end-stage diseases or disorders in which there is no other approach to therapy. Gene therapy may be considered for any disease where the potential of gene therapy is considered to be favorable relative to the risk, cost, and benefit of conventional therapeutics or other experimental therapies involving conventional drugs, advanced drug delivery methods, or biological products. With the development of increasingly safe and effective methods for vector delivery, it is likely that there will be clinical trials for a large number of inherited metabolic diseases rather than a select list of model disorders.
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