Cargando…
Assessment of AAV Dual Vector Safety in the Abca4(−)(/)(−) Mouse Model of Stargardt Disease
PURPOSE: Adeno-associated viral (AAV) gene therapy treatment for Stargardt disease currently requires a dual vector approach owing to the size of the ATP-binding cassette transporter family member gene (ABCA4). The nature of the dual vector system creates the potential for adverse events. Here we ha...
Autores principales: | McClements, Michelle E., Barnard, Alun R., Charbel Issa, Peter, MacLaren, Robert E. |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Association for Research in Vision and Ophthalmology
2020
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7115835/ https://www.ncbi.nlm.nih.gov/pubmed/32724727 http://dx.doi.org/10.1167/tvst.9.7.20 |
Ejemplares similares
-
Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes
por: McClements, Michelle E., et al.
Publicado: (2017) -
Therapy Approaches for Stargardt Disease
por: Piotter, Elena, et al.
Publicado: (2021) -
Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina
por: Patrício, Maria I., et al.
Publicado: (2017) -
Accurate Quantification of AAV Vector Genomes by Quantitative PCR
por: Martinez-Fernandez de la Camara, Cristina, et al.
Publicado: (2021) -
Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4(-/-) mouse and bipolar cells in the rd1 mouse and human retina ex-vivo
por: De Silva, Samantha R., et al.
Publicado: (2016)