Arbuthnot P, Maepa M B, Ely A, Pepper M S
Wits/SAMRC Antiviral Gene Therapy Research Unit, Department of Molecular Medicine and Haematology, School of Pathology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.
SAMRC Extramural Unit for Stem Cell Research and Therapy and Institute for Cellular and Molecular Medicine, Department of Immunology, Prinshof Campus, University of Pretoria, Pretoria, South Africa.
Gene Ther. 2017 Sep;24(9):581-589. doi: 10.1038/gt.2017.57. Epub 2017 Jul 10.
Gene therapy has made impressive recent progress and has potential for treating a wide range of diseases, many of which are important to Africa. However, as a result of lack of direct public funding and skilled personnel, direct research on gene therapy in Africa is currently limited and resources to support the endeavor are modest. A strength of the technology is that it is based on principles of rational design, and the tools of gene therapy are now highly versatile. For example gene silencing and gene editing may be used to disable viral genes for therapeutic purposes. Gene therapy may thus lead to cure from infections with HIV-1, hepatitis B virus and Ebola virus, which are of significant public health importance in Africa. Although enthusiasm for gene therapy is justified, significant challenges to implementing the technology remain. These include ensuring efficient delivery of therapeutic nucleic acids to target cells, limiting unintended effects, cost and complexity of treatment regimens. In addition, implementation of effective legislation that will govern gene therapy research will be a challenge. Nevertheless, it is an exciting prospect that gene therapy should soon reach the mainstream of medical management. Participation of African researchers in the exciting developments is currently limited, but their involvement is important to address health problems, develop capacity and enhance economic progress of the continent.
基因治疗最近取得了令人瞩目的进展，具有治疗多种疾病的潜力，其中许多疾病对非洲至关重要。然而，由于缺乏直接的公共资金和专业人才，目前非洲对基因治疗的直接研究有限，支持这项工作的资源也很有限。这项技术的一个优势在于它基于合理设计的原则，而且基因治疗工具现在具有很高的通用性。例如，基因沉默和基因编辑可用于治疗目的使病毒基因失活。基因治疗因此可能治愈在非洲具有重大公共卫生意义的HIV-1、乙型肝炎病毒和埃博拉病毒感染。尽管对基因治疗的热情是合理的，但实施这项技术仍面临重大挑战。这些挑战包括确保治疗性核酸有效递送至靶细胞、限制意外效应、治疗方案的成本和复杂性。此外，实施管理基因治疗研究的有效立法将是一项挑战。尽管如此，基因治疗很快应能进入医学管理主流是一个令人兴奋的前景。目前非洲研究人员对这些令人兴奋的进展参与有限，但他们的参与对于解决健康问题、培养能力和促进非洲大陆的经济发展至关重要。
