Final Term Lesson 4 - Gene Therapy

A promising future to disease treatment. A technique in correcting defective genes responsible for disease development. The introduction of genes to existing cells to prevent or cure a wide range of diseases. Therapy that is currently available primarily in a research setting.

• Defective genes
• Gene Therapy
• Nanotechnology
• GMO

The earliest method of gene therapy

• CRISPR-Cas9
• Gene Therapy
• Nanotechnology
• Gene transfer or Gene addition

A type of Gene Therapy wherein therapeutic genes transferred into somatic cells.

• In Vivo (inside your body)
• Ex Vivo (outside your body)
• Somatic Cell Gene Therapy
• Germ Line Gene Therapy

A type of Gene Therapy wherein therapeutic genes transferred into the germ cell.

• In Vivo (inside your body)
• Ex Vivo (outside your body)
• Somatic Cell Gene Therapy
• Germ Line Gene Therapy

Scientist put the vector carrying the new genes directly into your body via injection or intravenous (IV) infusion.

• In Vivo (inside your body)
• Ex Vivo (outside your body)
• Somatic Cell Gene Therapy
• Germ Line Gene Therapy

Scientist extract cells from your body and introduce them to the vector inside a petri dish, then your altered cell are returned to your body when they hopefully multiply.

• In Vivo (inside your body)
• Ex Vivo (outside your body)
• Somatic Cell Gene Therapy
• Germ Line Gene Therapy

inherited vision loss; blood disorders; Spinal muscular atrophy (SMA); Cerebral adrenoleukodystrophy (CALD); and Cancer

• True

• False

A newer technique that may potentially be used for gene therapy. Instead of adding new genetic material, genome editing introduces gene-editing tools that can change the existing DNA in the cell. It allows genetic material to be added, removed, or altered at precise locations in the genome. Introduces molecular tools to change the existing DNA in the cell.

• Genome editing
• Gene Therapy
• Nanotechnology
• GMO

A well-known type of genome editing.

• Gene Therapy
• Nanotechnology
• GMO
• CRISPR-Cas9

Genome editing is being studied to (choose 4):

• Fix a genetic alteration underlying a disorder, so the gene can function properly.
• Turn on a gene to help fight a disease.
• Uncontrollable population growth.
• Turn off a gene that is functioning improperly.
• Remove a piece of DNA that is impairing gene function and causing disease.