Pharmaceutical gene medicines for local and systemic therapy

Access through your institution Buy or subscribe Somatic gene therapy intends to provide specific cells of a patient with the _genetic software_ required to produce therapeutic proteins for

the prevention, modulation, or correction of a disease. One of the unique advantages of harnessing the patient's body to produce therapeutic proteins by administration of plasmid

therapeutics as pharmaceutical products (i.e., _gene medicines _) is to create a radically improved quality of pharmacological response for the prophylactic and therapeutic treatment of

genetic or acquired diseases. The opportunity of administering formulated plasmids, as pre-drugs, that use the patient's body to produce proteins in a natural, controllable,

cell-specific manner would introduce a new quality of medical treatment. The success of gene medicines will require the ability to control both the location and the functioning of an

administered gene in accessible tissues (e.g., skeletal muscle, solid tumors) in order to provide local or systemic effects (e.g., in cardiovascular diseases with angiogenic and

hematological factors, or in cancer treatment with immunotherapy and anti-angiogenesis). Cell-specific control of gene expression and drug-dependent regulation of gene expression (e.g., with

a GeneSwitch™) in vivo would also enable unique product opportunities. In addition, the systemic administration of formulated plasmid to target sites would create additional product

opportunities, for instance, in the treatment of disseminated tumors and metastases. This presentation describes some of the technologies and product concepts evaluated in animal models, and

gene medicines that are being evaluated in clinical trials for local and systemic gene therapy. This is a preview of subscription content, access via your institution ACCESS OPTIONS Access

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subscriptions * Read our FAQs * Contact customer support AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * Valentis, Inc., The Woodlands Center, 8301 New Trails Drive, The Woodlands, 77381, TX,

United States Alain Rolland Authors * Alain Rolland View author publications You can also search for this author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT

THIS ARTICLE CITE THIS ARTICLE Rolland, A. Pharmaceutical Gene Medicines for Local and Systemic Therapy. _Nat Biotechnol_ 17 (Suppl 4), 26 (1999). https://doi.org/10.1038/70151 Download

citation * Issue Date: December 1999 * DOI: https://doi.org/10.1038/70151 SHARE THIS ARTICLE Anyone you share the following link with will be able to read this content: Get shareable link

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