「생명윤리 및 안전에 관한 법률」유전자치료 조항에 대한 개선방안

김현철(Hyeon Cheol KIM),김휘원(Hwi Won KIM) 이화여자대학교 생명의료법연구소 2009 생명윤리정책연구 Vol.3 No.2

Due to the success of the treatment for ADA patient, gene therapy has been presented as a new treatment for serious diseases. Nowadays gene therapy is used more than 90 times a year around the world(2006). Though it has many benefits, as many studies has pointed out, it has various kinds of ethical problems, including its scientific uncertainty, clinical risks, and the potential violation of human dignity. The Bioethics and Safety Act has been established in order to address these problems in Korea in 2005. However two provisions relevant to gene therapy in the act are not regarded as proper measure to respond these problems. The aims of this paper is to review legal aspects of gene therapy in the Bioethics and Safety Act in 2008, and to find a way to improve the article of gene therapy in the act. First of all, vague and ambiguous expression of the definition of gene therapy should be crystallized into the definite one. Second, the gene therapy is in general prohibited, and in particular the gene therapy with respect to a spermatozoon, ovum, embryo or fetus is prohibited. There are three exceptional circumstances which allow gene therapy. The exceptional cases contain serious diseases such as hereditary diseases, cancers, and AIDS which may threaten life or cause any serious disability, and diseases whose gene therapy would be deemed considerably more effective than any other treatment. But, the range of these exceptions is too broad to regulate the use of gene therapy. Third, although gene therapy is not regarded as a tried and reliable remedy yet, the establishment and registration of gene therapy institution is easy and simple. The last, there are two kinds of laws relevant to gene therapy which are the Bioethics and Safety Act and Pharmaceutical Affairs Act. There are some inconsistency and confusions in implementing these two laws. This paper will address legal problems, articulate inconsistencies within these laws, and find better oversight system of gene therapy in Korea.

유전자치료법의 국가표준지침 제정에 관한 연구

남명진 국립보건연구원 1998 국립보건원보 Vol.35 No.-

「생명윤리 및 안전에 관한 법률」 유전자치료 조항에 대한 개선방안

김현철,김휘원 이화여자대학교 생명의료법연구소 2009 생명윤리정책연구 Vol.3 No.1

Due to the success of the treatment for ADA patient, gene therapy has been presented as a new treatment for serious diseases. Nowadays gene therapy is used more than 90 times a year around the world(2006). Though it has many benefits, as many studies has pointed out, it has various kinds of ethical problems, including its scientific uncertainty, clinical risks, and the potential violation of human dignity. The Bioethics and Safety Act has been established in order to address these problems in Korea in 2005. However two provisions relevant to gene therapy in the act are not regarded as proper measure to respond these problems. The aims of this paper is to review legal aspects of gene therapy in the Bioethics and Safety Act in 2008, and to find a way to improve the article of gene therapy in the act. First of all, vague and ambiguous expression of the definition of gene therapy should be crystallized into the definite one. Second, the gene therapy is in general prohibited, and in particular the gene therapy with respect to a spermatozoon, ovum, embryo or fetus is prohibited. There are three exceptional circumstances which allow gene therapy. The exceptional cases contain serious diseases such as hereditary diseases, cancers, and AIDS which may threaten life or cause any serious disability, and diseases whose gene therapy would be deemed considerably more effective than any other treatment. But, the range of these exceptions is too broad to regulate the use of gene therapy. Third, although gene therapy is not regarded as a tried and reliable remedy yet, the establishment and registration of gene therapy institution is easy and simple. The last, there are two kinds of laws relevant to gene therapy which are the Bioethics and Safety Act and Pharmaceutical Affairs Act. There are some inconsistency and confusions in implementing these two laws. This paper will address legal problems, articulate inconsistencies within these laws, and find better oversight system of gene therapy in Korea.

유전자 치료에 대한 윤리적 고찰

강미정(KANG Mi Jung) 한국생명윤리학회 2000 생명윤리 Vol.1 No.1

This article aims at an ethical review of gene therapy. For this purpose, I try to understand the ethical significance by categorizing it into three parts : somatic cell gene therapy, germ-line gene therapy, and gene manipulation for the improvement of gene function. First of all, somatic cell gene therapy has the general agreement that it has no ethical problems because its techniques are the same as blood transfusion or organ transplantation. Furthermore the somatic cell gene therapy can solve the problem of selective abortion. However, since we cannot ignore the possibility that the somatic cell gene therapy can control human's actions, we should limit this operation. Secondly, germ-line cell gene therapy is a hot issue. Some people support it because of its medical utilities, repect of the parent's autonomy, the right of freedom on scientific study, and the duty of improvement of the future generation. On the contrary, I analyzed that some are opposed to it because of its scientific uncertainty, clinical risks(hazard), the slippery slope on the improvement of human species, the uncertainty of the approvement by the future generation, the distribution of resources, and the interference on the whole genetical endowment. Thirdly, gene therapy is related to the improvement of human traits. In general this claim has many objections, such as playing God, the call for eugenics, the impossibility of value agreement in the view of cultural relativism. However, the development of genetic engineering is transferring the objections to voices of support. For this we should try to make counter-plans. One way to do this is to emphasize the responsibility on future generations. To be moral, we must release the future generation free from genetic diseases. And thus we should eliminate the origin of injustice and unequality. Futhermore, we have the responsibility to pass down good genes to the future generation. This is a mental gene that will make up a world of peace and love.

유전자편집 기술의 발전에 대응한 인간배아 유전자치료의 규제방향

박대웅(Park Dae-woong),류화신(Ryoo Hwa-shin) 한국생명윤리학회 2016 생명윤리 Vol.17 No.1

크리스퍼 유전자가위 기술은 본격적인 유전자치료 시대의 등장을 예고하고 있다. 특히 인간배아를 대상으로 하는 유전자치료는 세대 간에 이어지는 유전병의 고리를 끊을 수 있다는 엄청난 사회·경제적 효용을 가지고 있다. 하지만 인간으로 발생할 수 있는 배아의 연구 수단화, 기술적 한계로 인한 부작용, 유전자의 역할에 대한 지식의 한계, 결과가 발현될 다음 세대의 동의 부재, 치료가 아닌 강화에 적용될 가능성과 문제점 등 다양한 사회·윤리적 문제 역시 내포하고 있다. 이러한 문제점 때문에 대부분의 국가에서 인간배아를 대상으로 하는 유전자치료를 금지하고 있으나 그 잠재적 효용을 고려하여 치료를 위한 연구는 허용하는 국가들이 많다. 다가올 인간배아 유전자치료 시대를 대비한 규제방향에 대해서도 외국에서는 다양한 논의가 이루어지고 있다. 반면, 우리나라는 인간배아 유전자치료를 위한 연구의 허용 여부가 명확하지 않고 인간배아 유전자편집 행위에 대해서도 법률로 규율하고 있지 않다. 인간배아 유전자치료를 위한 연구는 위험성을 고려한 합리적 범위를 설정하여 허용하되, 생식 목적의 유전자편집 행위는 법률로 금지할 필요가 있다. 아울러 다가올 인간배아 유전자치료 시대에는 치료의 기술적·윤리적 한계선 설정, 안정성 확보를 위한 장기간의 추적조사, 경제력에 의존하지 않는 보편적 의료접근권 보장, 유전자치료 강제행위의 금지 등의 법제화가 필요할 것이다. The age of gene therapy is down the pike with the arrival of CRISPR-Cas9 enzyme scissors. Gene therapy on embryos can cause positive socio-economical impact in that it can break the cycle of genetic disorder. But there are various social and ethical issues, including using embryo for experiment, technological limitation, limit of knowledge about the function of genes, absent of consent by descendants, and using for genetic enhancement. For this reason, the reproductive use of edited embryos is prohibited in many countries. But scientific research are generally permitted considering the potential effectiveness. A regulatory framework for the age of gene therapy is also being discussed. But Korean law is ambiguous about whether scientific research for gene therapy on human embryo is permitted and there is no regulation for human embryos gene alteration. So scientific research for gene therapy on human embryo need to be permitted within reasonable limits, and human embryos gene alteration for reproductive purposes should be prohibited. And in human embryo gene therapy era, new regulations are necessary including technical and ethical limitation on therapy, multi generational follow-up for safety, access to therapy regardless of their economic power, and the prohibition of compulsory gene therapy.

Gene-Editing: Interpretation of Current Law and Legal Policy

Kim, Na-Kyoung The Korean Society of Developmental Biology 2017 발생과 생식 Vol.21 No.3

tWith the development of the third-generation gene scissors, CRISPR-Cas9, concerns are being raised about ethical and social repercussions of the new gene-editing technology. In this situation, this article explores the legislation and interpretation of the positive laws in South Korea. The BioAct does not specify and regulate 'gene editing' itself. However, assuming that genetic editing is used in the process of research and treatment, we can look to the specific details of the regulations for research on humans as well as gene therapy research in order to see how genetic editing is regulated under the BioAct. BioAct differentiates the regulation between (born) humans and embryos etc. and the regulation differ entirely in the manner and scope. Moreover, due to the fact that gene therapy products are regarded as drugs, they fall under different regulations. The Korean Pharmacopoeia Act put stringent sanctions on clinical trials for gene therapy products and the official Notification "Approval and Examination Regulations for Biological Products, etc." by Food and Drug Safety Administration may be applied to gene editing for gene therapy purposes.

Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy

Rajarathinam Thenmozhi,이지수,박나영,최병옥,홍영빈 한국뇌신경과학회 2020 Experimental Neurobiology Vol.29 No.3

Inherited peripheral neuropathy (IPN) is caused by heterogeneous genetic mutations in more than 100 genes. So far, several treatment options for IPN have been developed and clinically evaluated using small molecules. However, gene therapy-based therapeutic strategies have not been aggressively investigated, likely due to the complexities of inheritance in IPN. Indeed, because the majority of the causative mutations of IPN lead to gainof- function rather than loss-of-function, developing a therapeutic strategy is more difficult, especially considering gene therapy for genetic diseases began with the simple idea of replacing a defective gene with a functional copy. Recent advances in gene manipulation technology have brought novel approaches to gene therapy and its clinical application for IPN treatment. For example, in addition to the classically used gene replacement for mutant genes in recessively inherited IPN, other techniques including gene addition to modify the disease phenotype, modulations of target gene expression, and techniques to edit mutant genes have been developed and evaluated as potent therapeutic strategies for dominantly inherited IPN. In this review, the current status of gene therapy for IPN and future perspectives will be discussed.

줄기 세포 분야의 유전자 치료 연구 동향

오유경,정형민 한국약제학회 2002 Journal of Pharmaceutical Investigation Vol.32 No.2

Recently, stem cell-mediated gene therapy is emerging as a novel therapeutic approach. For the successful gene modification of stem cells, the development of a suitable gene transfer technique needs to be preceded. This review focuses on the various gene transfer techniques based on nonviral and viral vectors, and physical methods. The advantages and disadvantages of each gene transfer method are compared, and the general properties of these vectors are discussed in relation to the gene transfer in stem cell research. This review also highlights the therapeutic application of stem cell-mediated gene therapy. The choice of gene transfer vectors may vary depending on the type of the stem cells and the target of stem cell therapy. Of various gene transfer methods, viral vector-based gene therapy has been emphasized due to the higher transfection efficiency. The current status and up-to-date findings of stem cell-mediated gene therapy are discussed in the viewpoint of the various targets of stem cell therapy such as the modification of stem cell potency, the acceleration of regeneration process and the formation of expressional organization.

Molecular Imaging of Biological Gene Delivery Vehicles for Targeted Cancer Therapy: Beyond Viral Vectors

Min, Jung-Joon,Nguyen, Vu H.,Gambhir, Sanjiv S. The Korea Society of Nuclear Medicine 2010 핵의학 분자영상 Vol.44 No.1

Cancer persists as one of the most devastating diseases in the world. Problems including metastasis and tumor resistance to chemotherapy and radiotherapy have seriously limited the therapeutic effects of present clinical treatments. To overcome these limitations, cancer gene therapy has been developed over the last two decades for a broad spectrum of applications, from gene replacement and knockdown to vaccination, each with different requirements for gene delivery. So far, a number of genes and delivery vectors have been investigated, and significant progress has been made with several gene therapy modalities in clinical trials. Viral vectors and synthetic liposomes have emerged as the vehicles of choice for many applications. However, both have limitations and risks that restrict gene therapy applications, including the complexity of production, limited packaging capacity, and unfavorable immunological features. While continuing to improve these vectors, it is important to investigate other options, particularly nonviral biological agents such as bacteria, bacteriophages, and bacteria-like particles. Recently, many molecular imaging techniques for safe, repeated, and high-resolution in vivo imaging of gene expression have been employed to assess vector-mediated gene expression in living subjects. In this review, molecular imaging techniques for monitoring biological gene delivery vehicles are described, and the specific use of these methods at different steps is illustrated. Linking molecular imaging to gene therapy will eventually help to develop novel gene delivery vehicles for preclinical study and support the development of future human applications.

Gene Therapy : Basic Principles and Clinical Applications

Kim, Heyjin,Kim, Seung Taik 충북대학교 의과대학 충북대학교 의학연구소 2009 忠北醫大學術誌 Vol.18 No.2

1989년 인체에 retrovirus를 매개로 한 유전자요법이 처음 시도 된 이후로 유전자 요법은 그 적용범위를 광범하게 확장하고 있다. 특히, 유전질환 뿐만 아니라 암, 심혈관질환, 자가 면역질환으로 고통 받고 있는 환자들에게 유용하게 이용될 수 있는 치료방법으로 그 가능성을 지속적으로 검정해 나가고 있다. 유전자 치료의 핵심은 정상유전자를 세포에 이입시켜 발현시킴으로서 그 세포 또는 타 기관의 병적 상태를 교정하는 것이다. 따라서 정상유전자의 효과적인 이입방법, 대상세포의 선정이 중요하다. 이 논문에서는 바이러스를 이용한 유전자이입법과 비바이러스성 전달매개체를 이용한 두 가지 방법에 대하여 특징과 장, 단점을 알아보고, 현재 시도되고 있는 임상실험을 조명해 봄으로써 유전자요법의 현주소와 앞으로의 전망에 대해 논하고자 한다. Since 1989 when clinical trial of gene therapy using retroviral vector was done, gene therapy has widened its application. The possible role of gene therapy has been examined for cancer, cardiovascular diseases, and autoimmune diseases in addition to genetic disorders. The expression of intact protein in the cells through the transduction of normal gene into the cells is the central issue in gene therapy. In this point, the efficacy of gene transfer and the choice of target cells are very important. We reviewed here the characteristics of gene transfer methods, and discussed the prospect of gene therapy through the review of current clinical trials.