Choice of surrogate and physiological markers for prenatal gene therapy.
Surrogate genetically encoded markers have been utilized in order to analyze gene transfer efficacy, location, and persistence. These marker genes have greatly accelerated the development of gene transfer vectors for the ultimate application of gene therapy using therapeutic genes. They have also been used in many other applications, such as gene marking in order to study developmental cell lineages, to track cell migration, and to study tumor growth and metastasis. This chapter aims to describe the analysis of several commonly used marker genes: green fluorescent protein (GFP), β-galactosidase, firefly luciferase, human factor IX, and alkaline phosphatase. The merits and disadvantages of each are briefly discussed. In addition a few short examples are provided for continual and endpoint analysis in different disease models including hemophilia, cystic fibrosis, ornithine transcarbamylase deficiency and Gaucher disease.
Gene Transfer Technology Group, Institute for Women's Health, University College London, London, UK.
Juliette M K M
JM
Delhove
Ahad A
AA
Rahim
Tristan R
TR
McKay
Simon N
SN
Waddington
Suzanne M K
SM
Buckley
D000469:Alkaline Phosphatase / Q000378:metabolism; D000818:Animals; D015415:Biomarkers / Q000378:metabolism*; D004247:DNA / Q000302:isolation & purification; D057075:Enzyme Assays; D004797:Enzyme-Linked Immunosorbent Assay; D005164:Factor IX / Q000378:metabolism; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D049452:Green Fluorescent Proteins / Q000378:metabolism; D006801:Humans; D007150:Immunohistochemistry; D049409:Luciferases, Firefly / Q000378:metabolism; D051379:Mice; D016133:Polymerase Chain Reaction; D011295:Prenatal Care / Q000379:methods*; D013194:Staining and Labeling; D014020:Tissue Extracts; D051598:Whole Body Imaging; D001616:beta-Galactosidase / Q000378:metabolism
D015415:Biomarkers; D014020:Tissue Extracts; D049452:Green Fluorescent Proteins; D005164:Factor IX; D004247:DNA; D049409:Luciferases, Firefly; D000469:Alkaline Phosphatase; D001616:beta-Galactosidase