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Approaches to the treatment of genetic disease: lysosomal storage disorders (LSDs)

Published on August 31, 2025   32 min

Other Talks in the Series: Introduction to Human Genetics and Genomics

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0:00
Hello. My name is Paul Gissen, and I'm a professor of pediatric metabolic medicine at UCL Great Ormond Street Institute of Child Health. Today, I'm going to talk about the approaches to the treatment of genetic disease, specifically targeting the treatments that are gene-based. I target a little bit towards lysosomal storage disorders, which are more familiar to me.
0:30
What is gene therapy? Gene therapy is defined as genetic modification of somatic cells, and these include brain, liver, hematopoietic stem cells, muscle, and other cells too. There are different types of gene therapy. This could be gene addition, and this could be done by adding DNA or mRNA. This could be targeting transcription or, ultimately, genome editing. There are different methods of delivering gene therapy to the cells, and these delivery so-called vectors could be viral or non-viral, and sometimes some of the gene therapy platforms are using conjugated oligonucleotides.
1:18
In the next few slides, I'm going to talk about gene therapy vectors, both viral and non-viral. Let's start with the viral gene therapy vectors.
1:31
On the viral gene therapy vectors, the commonest vectors known are lentiviruses, adenoviruses and adeno-associated viruses.

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Approaches to the treatment of genetic disease: lysosomal storage disorders (LSDs)

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