Gene therapy Flashcards
(16 cards)
Definition of gene therapy.
function of the defective gene can be compensated by introducing or substituting the correct gene into the patient’s cell.
Result of having defective gene.
May lead to the absence of certain protein or the synthesis of non-functional proteins.
What are the types of gene therapy ?
(1) somatic cell gene therapy
(2) germ line gene therapy
Limitation of gene therapy
Only focus on single gene disorder
target gene of somatic cells
body cell
site of somatic cell gene therapy
in vivo, inside the body
effects on offspring of somatic cell gene therapy
Defective gene will be passed on to their offspring.
Since genetic material is unchanged in germ cells.
target gene of germ line gene therapy
(1) sex cells, gametes
(2) early embryonic cells
Effect on offspring by germ line cell gene therapy.
The genetic change will be inherited and pass to the next generation.
What is the process of gene therapy ?
(1) normal gene insert to vector
(2) recombinant vector infects and transfers the normal gene into target cell
(3) gene expression and protein synthesis
What can be vector in gene therapy ?
Virus or liposome .
Pathway of in vivo gene therapy
(1) normal gene
(2) vector
(3) target organ
Pathway of ex vivo gene therapy
(1) normal gene
(2) vector
(3) remove target cell out of body
(4) insert normal gene into target cell
(5) selection
(6) introduce target cell back to target organ
Pros of gene therapy
(1) cure genetic disorder
(2) prevent measure against disease
(3) treat cancer, heart disease, diabetes and infectious disease
Give an example of disease that can take preventive measure by gene therapy.
Parkinson.
Cons of gene therapy
(1) viral vector may cause disease again
(2) viral vectors may trigger immune response
(3) inappropriate gene expression
(4) no gene variation as there is germ line gene therapy
