| Title |
Pseudotyped AAV Vector-Mediated Gene Transfer in a Human Fetal Trachea Xenograft Model: Implications for In Utero Gene Therapy for Cystic Fibrosis
|
|---|---|
| Published in |
PLOS ONE, August 2012
|
| DOI | 10.1371/journal.pone.0043633 |
| Pubmed ID | |
| Authors |
Sundeep G. Keswani, Swathi Balaji, Louis Le, Alice Leung, Anna B. Katz, Foong-Yen Lim, Mounira Habli, Helen N. Jones, James M. Wilson, Timothy M. Crombleholme |
| Abstract |
Lung disease including airway infection and inflammation currently causes the majority of morbidities and mortalities associated with cystic fibrosis (CF), making the airway epithelium and the submucosal glands (SMG) novel target cells for gene therapy in CF. These target cells are relatively inaccessible to postnatal gene transfer limiting the success of gene therapy. Our previous work in a human-fetal trachea xenograft model suggests the potential benefit for treating CF in utero. In this study, we aim to validate adeno-associated virus serotype 2 (AAV2) gene transfer in a human fetal trachea xenograft model and to compare transduction efficiencies of pseudotyping AAV2 vectors in fetal xenografts and postnatal xenograft controls. |
X Demographics
The data shown below were collected from the profiles of 6 X users who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 6 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 5 | 83% |
| Scientists | 1 | 17% |
Mendeley readers
The data shown below were compiled from readership statistics for 36 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Spain | 1 | 3% |
| Unknown | 35 | 97% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Bachelor | 8 | 22% |
| Researcher | 7 | 19% |
| Student > Master | 6 | 17% |
| Professor > Associate Professor | 4 | 11% |
| Student > Ph. D. Student | 3 | 8% |
| Other | 6 | 17% |
| Unknown | 2 | 6% |
| Readers by discipline | Count | As % |
|---|---|---|
| Agricultural and Biological Sciences | 10 | 28% |
| Medicine and Dentistry | 8 | 22% |
| Biochemistry, Genetics and Molecular Biology | 4 | 11% |
| Neuroscience | 3 | 8% |
| Engineering | 2 | 6% |
| Other | 6 | 17% |
| Unknown | 3 | 8% |