Gene therapy
9 important questions on Gene therapy
What two types of gene therapy exist?
What three levels are there to treat genetic diseases? What are the three therapy categories?
Gene therapy, RNA therapy, drugs/compounds that influence protein
What is the aim of each level of therapy?
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?
What is interesting about genetics and human disease?
What is meant by repurposing drugs?
What is the most well known RNA treatment technique?
What is unique about cancer as a disease?
How does CAR-T work?
Or you produce a CAR, a specific receptor that recognises other types of tumour cell antigens
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