Journal
HUMAN GENETICS
Volume 141, Issue 5, Pages 1085-1091Publisher
SPRINGER
DOI: 10.1007/s00439-021-02399-5
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Funding
- Wellcome Centre Grant [091580/Z/10/Z]
- Wellcome Investigator Award [107930/Z/15/Z]
- European Research Council [EC 694295]
- Wellcome [109090]
- Wellcome Trust [107930/Z/15/Z, 091580/Z/10/Z] Funding Source: Wellcome Trust
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The review highlights the genetic basis of many neurological disorders and the efforts being made to develop genetic strategies for treating monogenic neurological disorders. It also addresses the challenges and ethical difficulties that may arise in the process.
In recent years, it has become increasingly apparent that many neurological disorders are underpinned by a genetic aetiology. This has resulted in considerable efforts to develop therapeutic strategies which can treat the disease-causing mutation, either by supplying a functional copy of the mutated gene or editing the genomic sequence. In this review, we will discuss the main genetic strategies which are currently being explored for the treatment of monogenic neurological disorders, as well as some of the challenges they face. In addition, we will address some of the ethical difficulties which may arise.
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