[New horizons for medicine on the basis of genetic technology]

In case of a single gene defect a number of appropriate gene probes are available for prenatal diagnosis. Knowledge of the genetic disorders enables in some cases 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 few patients have been treated in various indications. Difficult to be overcome are the low frequency and unspecific integration of inserted DNA into the chromosome, lack of sufficient transcription control and short half-lives 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 obligonucleotides 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 therapy with recombinant DNA derived human proteins is in therapeutic application to replace their counterparts from native source by 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 times frames can be expected allowing an early return on investment. The expected market potential for recombinant DNA derived pharmaceuticals in 1995 will reach 4.4 billion DM.