Lecture 19

Question 1

What are factors slowing gene therapy development?

Answer 1

• Multigene disorders
• Short-lived corrections
• Immune Response
• Off-target Effects
• Vector Problem

Question 2

What are the characteristics of Multigene Disorders?

Answer 2

• most common of diseases as opposed to rare, single gene disorders

Question 3

What are the characteristics of Short-Lived Corrections?

Answer 3

• achieved in most cases
• where non-stem cells are transformed, effects are limited
• multiple rounds of gene therapy are proposed

Question 4

What are the characteristics of Immune Response?

Answer 4

• always an issue with any delivery method

- memory makes multiple rounds of gene therapy difficult

Question 5

What are the characteristics of Off-Target Effects?

Answer 5

• An important issue where the gene therapy may:
  o Cause new mutations
  o Lead to genomic instability
  o Disrupt functioning/normal genes &
  pathways

Question 6

What are the characteristics of Vector Problems?

Answer 6

- Delivery of somatic cells (often not dividing) = major issue:
o Toxicity
o Immune Responses 
o Gene size & regulation
o Tissue specificity