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Gene therapy disease treatment

Exhibit 11.6 describes a recent gene therapy trial that resulted in unexpected outcomes, which the regulatory authorities have to consider. A recent report of gene therapy for treatment of Alzheimer s disease is also included. [Pg.367]

Mandel, R. J. et al. (1999a). Progress in direct striatal delivery of L-dopa via gene therapy for treatment of Parkinson s disease using recombinant adeno-associated viral vectors. Exp. Neurol. 159(1), 47-64. [Pg.219]

Successful application of ocular gene therapy has been demonstrated in animal diseases or animal models of human disease. The most encouraging results in the field of ocular gene therapy involve treatment of genetically inherited retinal degenerations occurring in various animal species. Conventional pharmacologic approaches... [Pg.163]

With recent developments in the field of gene therapy, diseases that for centuries have been considered hopeless are now potentially curable. Much of the therapy for these diseases is currently experimental. However, during the 21st century, physicians may be using genetic engineering techniques routinely for both the diagnosis and treatment of their patients. [Pg.206]

Although pharmacology still relies heavily on small chemical substances, biologicals like recombinant proteins, cell-based therapies, and gene-therapy approaches have great potential for the treatment of broad spectrum of diseases. Therefore, the focus of modem pharmacology should shift towards these innovative therapies. [Pg.532]

Pharmacogenomic studies are often carried out in the context of clinical trials which, by virtue of having both a treatment and placebo group, are able to assess the association between a gene and disease progression independent of therapy as well as interactive effects of genotypes and therapy on disease. Several of the phar-... [Pg.52]

DNA plasmid-based treatment ( gene therapy ) is considered an alternative to the one based on classical chemical drugs or proteins recovered from recombinant cells. Treatment of acquired and inherent genetic diseases as well as the use of DNA for the purpose of vaccination are potential applications of plasmid DNA (pDNA). The plasmid carries information that allows protein expression in the targeted human cells as well as eukaryotic regulatory elements and specific prokaryotic sequences that control replication in the host cell, see Fig. 10. Formulation is required for ex- or in-vivo administration. Selected systems for gene expression can be viral or non-viral. [Pg.77]

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures. [Pg.44]

Gene therapy is under study to determine whether it could be used to treat disease. Current research is evaluating the safety of gene therapy future studies will test whether it is an effective treatment option. Several studies have already shown that this approach can have very serious health risks, such as toxicity, inflammation, and cancer. Because the techniques are relatively new, some of the risks may be unpredictable however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research is as safe as possible. [Pg.45]

The first gene therapy trial was conducted in September 1990. A 4-year-old girl with SCID (an inherited immune disorder disease, otherwise known as the bubble boy syndrome) was treated in Cleveland, Ohio. She is doing well some 10 years after the treatment. A second girl with the same disorder underwent gene therapy and she too continues to do well. [Pg.126]

The potential contribution of stem cells to medical treatment lies in then-capability to differentiate and grow into normal, healthy cells. Using pluripotent stem cells, scientists are devising means to culture them in the laboratories and coax them to grow into various specialized cells. Rather than gene therapy, with stem cells we have the potential of cell therapy to repair our diseased tissues and organs. This will circumvent the lack of donor organs. Stem cells also provide the possibility for healthy cells to cure disabilities such as strokes, Parkinson s disease, and diabetes. [Pg.128]

Several gene therapies have received orphan drug designation these are treatments for cystic fibrosis, Gaucher disease, and metastatic brain tumor. [Pg.381]

Gene therapy is a long-established concept, described in 1970 as the use of recombinant DNA for the treatment or cure of inherited disease in man (1). In effect, the pathology of an inherited disorder can be alleviated by the expression of an introduced gene to compensate for the defective gene product. [Pg.294]

Currently, more than 400 human somatic cell gene therapy protocols are being tested. Most of these involve the use of genetically modified cells to treat noninherited diseases. For example, normal copies of the p53 tumor suppressor gene are inserted into lung tumors to halt tumor progression, and genetically modified cells have been used to create new coronary vessels in patients with coronary heart disease. Success has also been achieved in the treatment of hereditary disease (most notably, the recent successful treatment of X-linked severe combined immune deficiency see Clinical Correlate). [Pg.352]

Genetic engineering procedures are becoming more and more important in medicine for diagnostic purposes (A-C). New genetic approaches to the treatment of severe diseases are still in the developmental stage ( gene therapy, D). [Pg.264]

Gene engineering is the basis of gene therapy where genes are removed, replaced, or altered producing new proteins for the treatment of diseases such as muscular dystrophy, some cancers, adenosine deaminase deficiency, cystic fibrosis, and emphysema. [Pg.333]


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