Chapter title |
Small and Micro-Scale Recombinant Adeno-Associated Virus Production and Purification for Ocular Gene Therapy Applications
|
---|---|
Chapter number | 2 |
Book title |
Retinal Gene Therapy
|
Published in |
Methods in molecular biology, January 2018
|
DOI | 10.1007/978-1-4939-7522-8_2 |
Pubmed ID | |
Book ISBNs |
978-1-4939-7521-1, 978-1-4939-7522-8
|
Authors |
Christopher A. Reid, Daniel M. Lipinski |
Abstract |
Over the past two decades recombinant adeno-associated virus (rAAV) vectors have emerged as the gold standard for transferring genetic material to cells of the retina. The ability to effectively produce small batches of rAAV vector at high enough purity for in vitro and in vivo applications in a cost-effective manner is paramount. This is particularly the case when conducting preclinical experiments to screen novel serotypes, promoters or transgenes, where production of numerous vector batches is required. Current vector production methods often produce large quantities of vector, limiting the cost-effectiveness and practicality of such screening experiments, which often require only small volumes of vector to carry out. Herein, we describe a method to produce high titer (1012-1013 vector genomes (vg)/mL) rAAV vector on small (~100 μL) or micro (~15 μL) scale for in vitro and in vivo applications. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 34 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Researcher | 8 | 24% |
Student > Ph. D. Student | 6 | 18% |
Student > Doctoral Student | 3 | 9% |
Student > Bachelor | 2 | 6% |
Other | 2 | 6% |
Other | 4 | 12% |
Unknown | 9 | 26% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 12 | 35% |
Medicine and Dentistry | 5 | 15% |
Chemical Engineering | 2 | 6% |
Agricultural and Biological Sciences | 1 | 3% |
Pharmacology, Toxicology and Pharmaceutical Science | 1 | 3% |
Other | 2 | 6% |
Unknown | 11 | 32% |