Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

By F. Mingozzi, K. High. Nature reviews genetics, 2011

832 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/0b7571867f2cc73af5d5052f99143fff5a56ae7c

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Genetics

1. Jonathan Gootenberg
2. Eugene V. Koonin
3. Bernd Zetsche
4. Feng Zhang
5. Phillip A. Sharp
6. Xuebing Wu
7. Jennifer A. Doudna

In Molecular Biology

1. Feng Zhang
2. Jennifer A. Doudna
3. Jonathan Gootenberg
4. Eugene V. Koonin
5. Bernd Zetsche
6. Ophir Shalem
7. Phillip A. Sharp
8. David A. Scott

Citations in the corpus

• In vivo genome editing using Staphylococcus aureus Cas9 (2015, background): “For this purpose, adeno-associated virus (AAV) vectors are attractive vehicles 10 because of their low immunogenic potential, reduced oncogenic risk from host-genome integration 11 , and broad-range of serotype specificity 12–15 .”
• Immune responses to AAV vectors: overcoming barriers to successful gene therapy. (2013, neutral): “10,11 As one would predict, the in vivo administration of a viral vector necessitates grappling at close quarters with the human immune response to foreign antigens.”