Gene Therapy

5. A type of treatment in which a patient’s T cells (a type of immune system cell) are changed in the lab so they will attack cancer cells.
7. A revolutionary gene-editing tool used in gene therapy.
10. A type of virus that can be used as a vector in gene therapy.
11. A type of virus that can be used as a vector in gene therapy.
12. Disorders caused by the mutation in a single gene, often the target of gene therapy.
14. Disorders caused by the combined action of more than one gene, more challenging for gene therapy.
16. This controversial type of gene therapy involves changes to sperm or egg cells.
20. Stands for Transcription Activator-Like Effector Nucleases, another gene-editing tool.
21. A type of gene therapy where genes are changed in cells still in the body.
22. These are used to deliver genetic material into cells in gene therapy.
24. A fat-based vector used in gene therapy.
25. The gene that, when mutated, causes Cystic Fibrosis. It codes for a protein that regulates the movement of chloride and sodium ions across cell membranes.
26. The enzyme that acts like a pair of ‘molecular scissors’ in the CRISPR system.

1. A type of engineered DNA-binding protein used in gene editing.
2. A genetic disorder where blood does not clot normally, often treated with gene therapy.
3. A genetic disorder that has been a target for gene therapy.
4. This type of gene therapy targets cells that are not sperm or egg cells.
6. A type of gene therapy where cells are modified outside the body and then transplanted back in.
8. A type of retrovirus used as a vector in gene therapy.
9. An organ that can be affected by Cystic Fibrosis due to mutations in the CFTR gene.
13. The process of making precise and targeted modifications to the DNA of a cell.
15. A method of introducing DNA into cells using an electric field.
17. A method of introducing DNA into cells using a fine needle.
18. The first person to undergo successful gene therapy.
19. Short for Adenosine Deaminase Deficiency, a genetic disorder that Ashanti De Silva was treated for using gene therapy.
23. An abbreviation for Severe Combined Immunodeficiency, a genetic disorder that results in a weak immune system. It’s often a target for gene therapy, especially ADA-SCID which is caused by Adenosine Deaminase Deficiency.