11 theme: gene therapy Flashcards
Why gene therapies are performed?
To treat inherited and acquired genetic diseases - Cancer - AIDS / HIV
Name 3 single gene (monogenic) diseases?
Hemophilia
Cystic fibrosis
Hemoglobinopathy
Name polygenic disseases?
Hemophilia
Cystic fibrosis
Hemoglobinopathy
- How therapeutic genes are delivered to target cells to treat diseases? Simplify the principle.
Exogenous DNA (Therapeutic Gene): contains instructions how to make a protein that is defective (su defektu)
To introduce the DNA into the body, carriers, vectors are used (adenoviruses/Retrovirus/Lentivirus)
When introduced into the cell, production of therapeutic protein starts. Produced protein corrects the dissease.
What are gene therapy hazards?name 2
Mutagenesis
Inactivation of tumor suppressor gene ( which regulates cell growth), which can occur when therapeutic gene unintentionally disrupts a tumor suppressor gene.
What is Severe Combined Immunodeficiency (SCID)?
Fatal dysfunction of the immune system. Gene therapy can be used to heal it.
What is principle of action of chimeric antigenic receptor (CAR) for gene therapy?
It involves modifying a patient’s own T-cells (a type of immune cell) to express a synthetic receptor that enables T-cells to specifically recognize and attack cancer cells.
What are oncogenes?
Cancer inducing genes-they can mutate and make other genes cancerous.
What are ways to treat cancer?
Introducing tumor suppressor
Induction of apoptosis (cells overgo self-destruction)
Anti-angiogenic therapy (aim to “starve” the tumor of nutrients and oxygen, slowing or stopping its growth.)
Oncolytic viral therapy (modified viruses with defective genes)
What is angiogenesis?
the process by which new blood vessels are formed
For what neurodegenerative diseases gene therapy can be applied?
Alzheimers disease
Parkinson´ s disease (Loss of dopamine (neurotransmitter) producing cell))
