GENE THERAPY

Gene Therapy

A technique that uses a gene(s) to treat, prevent or cure a disease or medical disorder

Describe how gene therapy can prevent or cure a disease

• Adds new copies of a gene that is broken

• Replaces a defective or missing gene in a patient’s cells with a healthy version of that gene

Describe how genes are introduced into the body by direct delivery

• Vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells

Describe how genes are introduced into the body by cell-based delivery

• A sample of patient’s cells removed and exposed to vector in a laboratory

• The cells containing the vector are then returned to the patient

• If treatment is successful, new gene delivered by vector will make a functioning protein

CRISPR

A specific, efficient and versatile gene-editing technology that can be used to

modify, delete or correct precise regions of our DNA

Outline the uses of CRISPR

1. It can be used in gene therapy to target, remove or repair mutated or unwanted sections of DNA

2. It can also be used to sterilise disease causing parasites such as malaria

3. Also been adapted to turn genes on or off without altering their sequence

Describe advantages of altering the human genome

1. Useful in situations when both prospective parents are homozygous for a

disease-causing variant

• CRISPR can remove this faulty gene and prevent this couple from having children who will have the disease caused by this particular gene variant

1. Can also be used to treat cases of polygenic disorders

Discuss ethical concerns on altering the human genome

1. Edits may be made in the wrong place which will cause various different mutations and potential harmful effects - questions the safety of the process

2. Some researchers and bioethicists are concerned that any genome editing, even used for treating diseases and disorders, will lead to people using it for non-therapeutic and enhancement purposes - many view as controversial

3. Research on altering the human genome is conducted in viable or nonviable embryos leftover from IVF, or embryos created expressly for research - questions the morals of the process