Gene technology: chances for diagnosis and therapy.
In the case of a single gene defect, a number of appropriate gene probes are available for prenatal diagnosis. In some cases, knowledge of the genetic disorders enables early onset of therapy or the option for abortion. However, gene technology which enables the diagnosis should not be viewed from an ethical point of view but rather the action taken when diagnostic results are available. Gene therapy for a single gene defect still is at the early stage of development. Only a few patients have been treated in various indications. Difficult to overcome are the low frequency and unspecific integration of inserted DNA into the chromosome, lack of sufficient transcription control, and short half-life of the integrated gene. From an ethical perspective gene therapy complies with the therapeutic concept of medicine. Antisense oligonucleotides are under clinical development for blockage of the synthesis of oncogenes and viral proteins. Stability of oligonucleotides as well as selectivity for specific cells will have to be overcome for broader application. Its therapeutic application is in accordance with the ethical principles of medicine. Substitution therapies with recombinant DNA derived human proteins are in therapeutic application to replace their counterparts from native source in a safer way or for human pharmacologically active proteins which cannot be isolated from their natural source. For recombinant DNA derived proteins where the mode of action is known, short development time frames can be expected allowing for an early return on investment. The expected market potential for recombinant DNA derived pharmaceuticals in 1995 will reach 4,400 million DM. Due to their specificity, monoclonal antibodies are used for tumor imaging when labeled by 99mtechnetium or for tumor therapy when labeled by rhenium or yttrium. Both concepts are under clinical evaluation. Vaccines derived from recombinant DNA technology offer the chance of producing safer vaccines consisting of the antigen determinant only. In general, recombinant DNA technology and biotechnology offer the opportunity of providing new diagnostic and therapeutic principles of high ethical value. The biotechnical manufacturing processes used for this purpose are friendly to the environment by using raw material from renewable sources, low energy consumption, and producing biodegradable products only. In almost all cases, host cells used for manufacturing belong to the safety category 1, in which no danger is expected for the operator, the public, and the environment.(ABSTRACT TRUNCATED AT 400 WORDS)
Werner
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Dr. Karl Thomae GmbH, Department Biotechnical Production, Biberach an der Riss, Germany.
D001702:Biopharmaceutics / Q000379:methods; D001709:Biotechnology / Q000639:trends; D002986:Clinical Trials as Topic; D005260:Female; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009693:Nucleic Acid Hybridization; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use; D011247:Pregnancy; D011296:Prenatal Diagnosis / Q000379:methods*
D016376:Oligonucleotides, Antisense