Gene Interventions Flashcards
What are diseases
Abnormal condition that adversely affects the structure or function of an organism.
These can be due to external factors like pathogens or inherited.
Why it can be hard to detect diseases
Diseases can be complex being caused by a combination of genetic and environmental factors.
It can require extensive genetic testing and then interpretation which then can raise ethical and social concerns on consent and privacy of results.
Give examples on how genetic material may be delivered
Viral vectors
Highly efficient at delivering genetic material into target cells for long-term expression.
There are safety concerns related to the potential for viral vectors to induce an immune response or insertional mutagenesis.
Best Used in the treatment of genetic disorders.
Non-Viral Vectors (e.g., Liposomes, Naked DNA, Gene Gun)
Non-viral vectors are generally safer and less immunogenic than viral vectors. They can accommodate larger genetic material and are more cost- effective to produce.
But less efficient at gene delivery and may not achieve long-term gene expression.
Best Used where transient gene expression is sufficient, such as in cancer gene therapy or vaccination.
What is in Vivo gene therapy
New or therapeutic genes are injected directly into patients body using viral vector and genetic material is delivered to target cells.
Well suited for ocular disorders where structure/function of cells has been altered.
What is ex Vivo gene therapy
Removal of specific cells form a patient which are then modified and then transplanted back into patient.
Applied to hematopoietic stem cells and is used to treat blood diseases.
