[Somatic gene therapy. Initial clinical applications and current research approach].
Somatic gene therapy represents an attempt to cure the genetic defects responsible for metabolic and neurological diseases, a major application is the treatment of cancer. Phase I studies have already been implemented. Although advances have been made, a number of central questions have yet to be answered before somatic gene therapy can be applied safely and effectively in patients. The development of effective techniques in gene transfer and cell transplantation is essential for successful therapy. Since this is a new area of research and development, and since genetically inherited diseases differ widely, many strategies are currently tested in animal models. Some of these strategies are discussed in the present review.
Runge
D M
DM
Department of Pathology, University of Pittsburgh.
Runge
D
D
D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics; D000256:Adenoviridae / Q000235:genetics; D000328:Adult; D000818:Animals; D002648:Child; D018014:Gene Transfer Techniques; D030342:Genetic Diseases, Inborn / Q000235:genetics* / Q000628:therapy; D015316:Genetic Therapy; D006467:Hemophilia A / Q000235:genetics / Q000628:therapy; D002836:Hemophilia B / Q000235:genetics / Q000628:therapy; D006801:Humans; D010661:Phenylketonurias / Q000235:genetics / Q000628:therapy
D000243:Adenosine Deaminase
Somatische Gentherapie. Erste klinische Anwendungen und heutige Forschungsansätze.