Researchers from Scripps Research have developed a new method to control and modulate gene therapy dosing. The researchers used a molecular switch that could be deployed towards controlling dosing in gene therapies. The relevance of gene therapy in the contemporary times cannot be undermined. For this reason, any form of developments in gene techniques holds immense significance for doctors and medical professionals. The researchers have proposed the first technique that could alter therapeutic gene activity. Moreover, these techniques could be useful in sound treatment of genetic disorders. The findings of the research are published in Nature Biotechnology journal. The research opens new avenues for addressing safety issues related to gene therapy.
Developing Molecular Switches
The researchers demonstrated their technique by embedding the switch into gene therapy that produces erythropoietin hormone. The secretion of this hormone helps in treatment of anaemia. The treatment line proposed by Scripps researchers involves the use of morpholinos, a control molecule approved by the Food and Drug Administration (FDA). The researchers first suppressed the expression of the gene at hand, and successively enhanced its expression over a wide range. The researchers consider this technique to be the only viable option in regulating dosing in gene therapy. Use of therapeutic gene copies inserted in the cells of patients is a widely used technique within gene therapy. The technique endows immense potential to cure diseases related to impaired genes.
Added Advantages of Transgene Therapy
The new technique, known as transgene switching system, could be used in an array of gene therapies. Furthermore, approval of morpholines by FDA gives premium value to the research. The team of researchers used ribozymes for the development of a transgene switch. The next decade is expected to witness several improvements in gene therapy and transgene methodologies.