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
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Professor Francesco Muntoni, University College London.

Date: 3rd February 2017
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Dr Tassos Georgiadis, Senior Research Associate, UCL Institute of Ophthalmology.

Date: 3rd February 2017
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Professor Waseem Qasim, Professor of Cell and Gene Therapy, University College London Institute of Child Health.

Date: 3rd February 2017
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Dr Ben Davies, Head of Genome Engineering Core Facility, Wellcome Trust Centre for Human Genetics

Date: 3rd February 2017
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key references 

All the references listed below are available in the RSM Library collection and can be accessed via the RSM’s e-resources area.

external references 

The following references are not available in the RSM Library; however, they can be accessed online via external websites.