Gene therapy Flashcards
what is gene therapy?
Treatment method based on the delivery of nucleic acids to cells (transfection) to treat a medical condition
what is transfection?
a procedure that introduces foreign nucleic acids into cells to produce genetically modified cells
how can gene therapy be used?
▫ Introduce a new gene OR Replace a defected/mutated gene
= using a Plasmid DNA
▫ Silence a gene that is not working properly ( STOP protein expression)
= Small interfering RNA (siRNA) OR Short-Hairpin RNA (shRNA)
when are gene therapy used to treat patients?
- single gene defect diseases (genetic/ hereditary)
- poly-genetic or non-hereditary disease
what are some examples of single gene defect diseases (genetic/ hereditary) used in gene therapy?
haemophilia
what are some examples of poly-genetic or non-hereditary disease used in gene therapy?
- cancer
- cvd
- hepatitis
what is poly-genetic or non-hereditary disease?
more than one gene/ environmental factors involved, harder to treat
how does SARS-COV-2 vaccines prevent disease?
using gene therapy to provide antibodies to target the spike proteins
providing an immune response
what are the different techniques of gene therapy transfection methods?
in-vivo therapy (increase risk)
ex-vivo therapy
how do you transfer genetic material?
non-viral delivery vectors (synthetic)
-liposomes
-CNTs
viral delivery vectors
how do viruses work?
two viral cycles - lytic and lysogenic
explain the two viral cycles
what are lytic viruses?
virus enters host,
replicates, and lyses
(burst open) occurs
causing death of host cell
immediately
what are lysogenic/latent viruses?
Some viruses have the ability to become dormant inside the cell
-They are called latent viruses
-They may remain inactive for
long periods of time (years)
Later, they activate to produce
new viruses in response to some
external signal
what are some examples of lysogenic/latent viruses?
- HIV and Herpes viruses