HG exam 3 pgs 31-35

Gene Therapy

Introducing cloned genes into living cells to treat disease.

SCID

Severe Combined Immunodeficiency, first disease treated by gene therapy in 1989.

Kymriah

First FDA-approved gene therapy for acute lymphoblastic leukemia on August 30, 2017.

Yescarta

FDA-approved gene therapy for non-Hodgkin lymphoma on October 18, 2017.

In-vivo gene delivery

Gene delivered directly into the patient.

Ex-vivo gene delivery

Cells taken out of the patient and modified before being placed back.

Viral vectors

Used to deliver genes, including retroviruses, adenoviruses, and lentiviruses.

Retroviruses

Viral vectors that can only infect dividing cells and can integrate into the host genome.

Adenoviruses

Viral vectors that do not integrate into the host genome and can carry medium-sized genes.

Lentiviruses

A type of retrovirus that can infect dividing and non-dividing cells and can carry large genes.

Non-viral vectors

Methods of gene delivery that do not use viruses, such as liposomes and nanoparticles.

Gene Pill

A method of delivering DNA therapeutically through ingestion.

RNA interference (RNAi)

A method to inhibit mRNA of defective genes to prevent their expression.

Induced pluripotent stem cells (iPS cells)

Adult cells reprogrammed to become pluripotent, similar to embryonic stem cells.

Allogenic cell therapy

Stem cell therapy using cells from a donor.

Autologous cell therapy

Stem cell therapy using the patient's own cells to avoid rejection.

Mesenchymal stem cells

Stem cells found throughout the body capable of becoming various cell types.

Hemopoietic stem cells

Stem cells used in bone marrow transplants to treat blood cancers.

Clinical trial

A research study to evaluate the safety and effectiveness of a new treatment.

Experimental intervention

A new or untested treatment that has not been proven safe or effective.

CRISPR-cas 9

A genome editing technology that allows for specific changes to DNA.