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Chapter title |
Retinal Gene Therapy for Choroideremia: In Vitro Testing for Gene Augmentation Using an Adeno-Associated Viral (AAV) Vector
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Chapter number | 7 |
Book title |
Retinal Gene Therapy
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Published in |
Methods in molecular biology, January 2018
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DOI | 10.1007/978-1-4939-7522-8_7 |
Pubmed ID | |
Book ISBNs |
978-1-4939-7521-1, 978-1-4939-7522-8
|
Authors |
Maria I. Patrício, Robert E. MacLaren |
Abstract |
As gene therapy of choroideremia is becoming a clinical reality, there is a need for reliable and sensitive assays to determine the expression of exogenously delivered Rab Escort Protein-1 (REP1), in particular to test new gene therapy vectors and as a quality control screen for clinical vector stocks. Here we describe an in vitro protocol to test transgene expression following AAV2/2-REP1 transduction of a human cell line. Gene augmentation can be confirmed by western blot and quantification of the fold-increase of human REP1 levels over untransduced controls. |
Mendeley readers
The data shown below were compiled from readership statistics for 6 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 6 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Bachelor | 2 | 33% |
Unspecified | 1 | 17% |
Researcher | 1 | 17% |
Other | 1 | 17% |
Unknown | 1 | 17% |
Readers by discipline | Count | As % |
---|---|---|
Agricultural and Biological Sciences | 2 | 33% |
Biochemistry, Genetics and Molecular Biology | 2 | 33% |
Unspecified | 1 | 17% |
Unknown | 1 | 17% |