gene-cell-therapy

Gene Therapy is the transfer of genetic material (gene transfer) to dysfunctional cells to correct a deficiency in the DNA or genome of a patient. Cell Therapy, such as stem cell therapy, can also be used to achieve these goals. These approaches can be applied to genetic disorders as well as diseases acquired over the lifetime of an individual, such as cancer or infection, to confer a specific property to the cell allowing it to combat the disease.

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Gene Therapy in the News

  • Early indicators of the malaria parasite in Africa developing resistance to the most effective drug available have been confirmed, according to new research. Investigators found Plasmodium falciparum malaria parasites with a mutation to the gene Ap2mu were less sensitive to the antimalarial drug artemisinin.

  • Children with 'bubble boy disease' who undergo gene therapy have fewer infections and hospitalizations than those receiving stem cells from a partially matched donor, new research suggests. Children with SCID-X1 are born with a genetic defect that prevents them from developing a normal immune system. Because they are prone to life-threatening infections, infants with SCID-X1 must be kept in a sterile, protective bubble and require extensive treatment for survival beyond infancy.

  • Researchers have developed a new method to activate genes by synthetically creating a key component of the epigenome that controls how our genes are expressed. The new technology allows researchers to turn on specific gene promoters and enhancers -- pieces of our genomes that control our gene activity -- by chemically manipulating proteins that package our DNA.

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© 2012 Gene Therapy Review. All Rights Reserved. ISSN (print) 1792-0094, ISSN (online) 1792-0108