Chapter title |
Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing
|
---|---|
Chapter number | 2 |
Book title |
Precision Medicine, CRISPR, and Genome Engineering
|
Published in |
Advances in experimental medicine and biology, January 2017
|
DOI | 10.1007/978-3-319-63904-8_2 |
Pubmed ID | |
Book ISBNs |
978-3-31-963903-1, 978-3-31-963904-8
|
Authors |
Benjamin E. Epstein, David V. Schaffer |
Abstract |
With the recent advent of several generations of targeted DNA nucleases, most recently CRISPR/Cas9, genome editing has become broadly accessible across the biomedical community. Importantly, the capacity of these nucleases to modify specific genomic loci associated with human disease could render new classes of genetic disease, including autosomal dominant or even idiopathic disease, accessible to gene therapy. In parallel, the emergence of adeno-associated virus (AAV) as a clinically important vector raises the possibility of integrating these two technologies towards the development of gene editing therapies. Though clear challenges exist, numerous proof-of-concept studies in preclinical models offer exciting promise for the future of gene therapy. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Russia | 1 | 33% |
Netherlands | 1 | 33% |
Unknown | 1 | 33% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 2 | 67% |
Scientists | 1 | 33% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 54 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Master | 11 | 20% |
Student > Bachelor | 10 | 19% |
Student > Ph. D. Student | 7 | 13% |
Researcher | 6 | 11% |
Other | 3 | 6% |
Other | 6 | 11% |
Unknown | 11 | 20% |
Readers by discipline | Count | As % |
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Biochemistry, Genetics and Molecular Biology | 18 | 33% |
Engineering | 6 | 11% |
Agricultural and Biological Sciences | 4 | 7% |
Immunology and Microbiology | 3 | 6% |
Medicine and Dentistry | 3 | 6% |
Other | 7 | 13% |
Unknown | 13 | 24% |