Resources: An overview of the use of gene editing in medicine
Gene editing is a technique that can be used to modify precisely DNA within a cell. Using a ‘find and replace’ strategy, it involves making a ‘cut’ at a specific DNA sequence, then removing it or replacing it with the desired one.
Emerging therapeutic applications include the ability to treat certain diseases such as leukaemia, and rare genetic disorders like Duchenne muscular dystrophy. It also has the potential to alter the genetic code of human embryos under strict regulatory conditions.
This resource page provides an overview of gene editing, highlighting how it can be applied to certain conditions and rare genetic disorders. It also explores its potential use in reproductive technologies and the ethical concerns surrounding this.
Lectures and key references focus on the use of gene editing in the following areas:
- Muscular dystrophy
- Childhood leukaemia
- Genetic retinal dystrophy
- Human reproductive research
Professor Francesco Muntoni, University College London.Date: 3rd February 2017
Dr Tassos Georgiadis, Senior Research Associate, UCL Institute of Ophthalmology.Date: 3rd February 2017
Professor Waseem Qasim, Professor of Cell and Gene Therapy, University College London Institute of Child Health.Date: 3rd February 2017
Dr Ben Davies, Head of Genome Engineering Core Facility, Wellcome Trust Centre for Human GeneticsDate: 3rd February 2017
All the references listed below are available in the RSM Library collection and can be accessed via the RSM’s e-resources area.
- Bakondi, B., Lv, W., Lu, B., Jones, M.K., Tsai, Y., Kim, K.J., Levy, R., Akhtar, A.A., Breunig, J.J., Svendsem, C.N., and Wang, S. (2016). In vivo CRISPR/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of autosomal dominant retinitis pigmentosa. Molecular Therapy, 24(3), pp. 556-563.
- Gilham, D.E., Anderson, J., Bridgeman, J.S., Hawkins, R.E., Exley, M.A., Stauss, H., Maher, J., Pule, M., Sewell, A.K., Bendle, G., Lee, S., Qasim, W., Thrasher, A., and Morris, E. (2015). Adoptive T-cell therapy for cancer in the United Kingdom: a review of activity for the British Society of Gene and Cell Therapy annual meeting 2015. Human Gene Therapy, 26(5), pp. 276-85.
- Hollinger, K., and Chamberlain, J.S. (2015). Viral vector-mediated gene therapies. Current Opinion in Neurology, 28(5), pp. 522-527.
The following references are not available in the RSM Library; however, they can be accessed online via external websites.
- Bakondi, B. (2016). In vivo versus ex vivo CRISPR therapies for retinal dystrophy. Expert Review of Ophthalmology, 11(6), pp. 397-400.
Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5289756/pdf/nihms-838298.pdf
- Camporesi, S., and Cavaliere, G. (2016). Emerging ethical perspectives in the clustered regularly interspaced short palindromic repeats genome-editing debate. Personalized Medicine, 13(6), pp. 575-586.
Available at: http://www.futuremedicine.com/doi/full/10.2217/pme-2016-0047
- Ge, X., Xi, H., Yang, F., Zhi, X., Fu, Y., Chen, D., Xu, R., Lin, G., Qu, J., Zhao, J., and Gu, F. (2016). CRISPR/Cas9-AAV mediated knock-in at NRL locus in human embryonic stem cells. Molecular Therapy. Nucleic Acids, 5(11), pp. e393.
Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5155318/pdf/mtna2016100a.pdf
- Gross, M. (2015). Bacterial scissors to edit human embryos? Current Biology: CB, 25(11), pp. R439-R442.
Available at: http://www.sciencedirect.com/science/article/pii/S0960982215006016
- Hu, J., Xia, E., Yang, L., and Xiao, X. (2016). Gene editing: A new step and a new direction toward finding a cure for Duchenne muscular dystrophy (DMD). Genes and Diseases, 3(2), pp. 101-102.
Available at: http://www.sciencedirect.com/science/article/pii/S2352304216000076
- Vassena, R., Heindryckx, B., Peco, R., Pennings, G., Raya, A., Sermon, K., and Veiga, A. (2016). Genome engineering through CRISPR/Cas9 technology in the human germline and pluripotent stem cells. Human Reproduction Update, 22(4), pp. 411-419.
Available at: https://academic.oup.com/humupd/article-lookup/doi/10.1093/humupd/dmw005
- Yanik, M., Müller, B., Song, F., Gall, J., Wagner, F., Wende, W., Lorenz, B., and Stieger, K. (2017). In vivo genome editing as a potential treatment strategy for inherited retinal dystrophies. Progress in Retinal and Eye Research, 56, pp. 1-18.
Available at: http://www.sciencedirect.com/science/article/pii/S1350946216300441