Viral Vector Gene therapy

Question 1

4 pros of viral vector gene therapy

Answer 1

Efficient gene delivery
Targeted cell types from tropisms
Potential cure for genetic disorders
Potent

Question 2

5 cons of viral vector gene therapy

Answer 2

Immunologic responses
Failure of gene therapy
Oncogenesis
Neutralising AAV antibodies
Insertional mutagenesis

Question 3

Example of an immunologic response to viral vector gene therapy

Answer 3

Induces innate and adaptive immune response
May limit haemophilia gene therapy efficacy

Question 4

Example of failure of gene therapy

Answer 4

Initial trials of haemophilia therapies,. still no approved therapy for A

Question 5

Oncogenesis viral vector gene therapy example

Answer 5

IV injection of AAV into mice caused development of HCC in 33% of mice

Question 6

Example of pre existing anti AAV antibodies

Answer 6

20% of haemophilia patients have anti-AAV antibodies