Animation from PBS Online: Cytoplasm transfer

Animation from PBS Online: Viral transduction

Bubble boy - the movie "Premise: A young man who has lived his whole life in a bubble in his bedroom (Gyllenhaal) decides to venture out into the world when he finds out that his childhood sweetheart (Shelton) is engaged to another man. So, he builds a portable bubble, and sets forth to get to her before her wedding at Niagara Falls. There's a 100% likelihood of an outbreak of hilarity and likely high jinks. (Kurtz plays the Bubble Boy's mother). " http://www.upcomingmovies.com/bubbleboy.html

http://www.rottentomatoes.com/m/TheBoyinthePlasticBubble-1002981/about.php

John Travolta - "The boy in the plastic bubble"

Bubble boy - in reality

Examples

• replacement of a cell surface receptor
• replacement of an intracellular enzyme
• injection of cells modified to secrete a factor
• suicide vectors

http://www.hosppract.com/genetics/graphics/9910mmc1.gif

A brief overviewfrom Biology 572

Overview of gene therapy - Hospital practice

Reference readings

Gene Therapy (Oak Ridge National Labs)

Hurdles:

• gene delivery tool.
• understanding gene function
• multigene disorders

Trials - Charts and statistics on current trials, from Journal of Gene Medicine

Clinical results

Interactive database

• X-linked severe combined immunodeficiency
• ADA deficiency (ADA-SCID)
  • PBS online
• Mucopolysaccharidosis
• Familial hypercholesterolemia
• Cystic fibrosis
• Hemophilia B (factor IX deficiency)
• Chronic granulomatous disease (p47phox deficiency)

Delivery methods and vectors

Retroviruses

Retroviral vectors

Adenoviruses

Human adenovirus vectors

Mouse adenovirus

Adenoassociated viruses

AAV

Herpes Simplex viruses

HSV

Epstein Barr virus episome in an HSV coat

Liposomes