Werner R G
Dr. Karl Thomae GmbH, A Biotechnische Produktion, Biberach an der Riss.
Arzneimittelforschung. 1995 Sep;45(9):1040-7.
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.
对于单基因缺陷,有多种合适的基因探针可用于产前诊断。了解遗传疾病在某些情况下能够使治疗尽早开始或提供堕胎选择。然而,能够进行诊断的基因技术不应从伦理角度看待,而应从获得诊断结果后采取的行动来考虑。针对单基因缺陷的基因治疗仍处于发展的早期阶段。仅有少数患者在各种适应症下接受了治疗。难以克服的问题包括插入的DNA在染色体中的整合频率低且不具有特异性、缺乏足够的转录控制以及整合基因的半衰期短。从伦理角度来看,基因治疗符合医学的治疗理念。反义寡核苷酸正处于临床开发阶段,用于阻断癌基因和病毒蛋白的合成。为了更广泛地应用,必须克服寡核苷酸的稳定性以及对特定细胞的选择性问题。其治疗应用符合医学的伦理原则。用重组DNA衍生的人蛋白进行替代疗法正在临床应用中,以用人源药理活性蛋白替代天然来源的相应蛋白,而这些天然来源的蛋白无法从其天然来源中分离出来。对于作用方式已知的重组DNA衍生蛋白,预计开发时间框架较短,能够早日实现投资回报。1995年重组DNA衍生药物的预期市场潜力将达到44亿德国马克。