Gene Therapy

Question 1

Adeno-Associated Virus (AAV)

Answer 1

Single stranded DNA genome of virus that infects host –> upon replication forms double stranded circular genome

By removing helper viruses in gene therapy that allow virus propagation, you can engineer cDNA encoding proteins in diseases like SMA (SMN1) under control of a promoter in hepatic cells w/o killing cells.

Question 2

Retrovirus

Answer 2

Virus infects cell w/ RNA genome, used as template to make DNA strand –> integrate dsDNA into genome and express gene of interest w/o virus completing its lifecycle and actually infecting patients

Question 3

CAR T-Cell Therapy

Answer 3

Infect T-Cell with modified HIV encoding CAR receptor –> recognizes B cell receptor CD19 in ALL and targets / kills B cells w/o infecting the host.

Question 4

CRISPR-Cas9

Answer 4

Intentionally causes a dsDNA break using gRNA complimentary to region of genome you want to edit, can knock in or knock out target genes.