Cells/Organs Inherited Disorders Flashcards
1
Q
Haemopoietic system disorders
A
Adenosine deaminase deficiency & Haemaglobinopathies
2
Q
Secretory epithelium disorder
A
cystic fibrosis
3
Q
Muscle cells
A
Muscular Dystrophy
4
Q
Nerve cells
A
Neurodegenerative disorders
5
Q
For inherited disorders in gene therapy…
A
Stable integration into host genome or sustained episomal expression of vector is required
6
Q
For cancers in gene therapy…
A
Transient expression is sufficient as short-term expression of a therapeutic gene should suffice
