Gene therapy

9 important questions on Gene therapy

What two types of gene therapy exist?

Treatment of genetic disease and genetic treatment of disease

What three levels are there to treat genetic diseases? What are the three therapy categories?

Gene, mRNA and protein level
Gene therapy, RNA therapy, drugs/compounds that influence protein

What is the aim of each level of therapy?

Protein: normalise protein function
RNA: normalising RNA levels, or correcting abnormal splicing with antisense oligo's
DNA: correcting mutation
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What level of therapy is the best?

DNA is perfect, since it's a full cure, other levels mean multiple treatments or a pill every day

What is interesting about genetics and human disease?

Humans are the animal model here, to find what mutations are causal for the disease. These can be built into zebrafish to try and find a possible cure

What is meant by repurposing drugs?

Looking at compounds that are already allowed and finding out whether these can also work to cure another disease

What is the most well known RNA treatment technique?

RNAi: short signalling RNA's that inhibit translation by causing cleavage or transcriptional silencing

What is unique about cancer as a disease?

The 'toxic' part is the tumour cell itself

How does CAR-T work?

A form of immune therapy, you either produce a T-cell receptor (TCR) that can target tumour using MHC.
Or you produce a CAR, a specific receptor that recognises other types of tumour cell antigens

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