Cargando…

Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues

Mostrar otras versiones (1)

The therapeutic effects of gene therapy using adeno-associated virus (AAV) vectors are dependent on the efficacy of viral transduction. Currently, we have reached the safe limits of AAV vector dose, beyond which damaging inflammatory responses are seen. To improve the efficacy of AAV transduction, w...

Descripción completa

Detalles Bibliográficos
Autores principales:	Chandler, Laurel C., Barnard, Alun R., Caddy, Sarah L., Patrício, Maria I., McClements, Michelle E., Fu, Howell, Rada, Cristina, MacLaren, Robert E., Xue, Kanmin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6606965/ https://www.ncbi.nlm.nih.gov/pubmed/31309129 http://dx.doi.org/10.1016/j.omtm.2019.05.012

Ejemplares similares

Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues
por: Chandler, Laurel C., et al.
Publicado: (2023)

Immunomodulatory Effects of Hydroxychloroquine and Chloroquine in Viral Infections and Their Potential Application in Retinal Gene Therapy
por: Chandler, Laurel C., et al.
Publicado: (2020)

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
por: Chandler, Laurel C., et al.
Publicado: (2021)

 Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes
por: McClements, Michelle E., et al.
Publicado: (2017)

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)

Assessment of AAV Dual Vector Safety in the Abca4(−)(/)(−) Mouse Model of Stargardt Disease
por: McClements, Michelle E., et al.
Publicado: (2020)

RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences
por: Fry, Lewis E., et al.
Publicado: (2020)

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
por: Han, Ruofan Connie, et al.
Publicado: (2021)

Genome-Editing Strategies for Treating Human Retinal Degenerations
por: Quinn, Joel, et al.
Publicado: (2021)

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
por: Orlans, Harry O., et al.
Publicado: (2021)

Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells
por: Dreismann, Anna K., et al.
Publicado: (2021)

Analysis of Early Cone Dysfunction in an In Vivo Model of Rod-Cone Dystrophy
por: Hassall, Mark M., et al.
Publicado: (2020)

CRISPR-Cas9 DNA Base-Editing and Prime-Editing
por: Kantor, Ariel, et al.
Publicado: (2020)

Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina
por: Salman, Ahmed, et al.
Publicado: (2021)

Therapy Approaches for Stargardt Disease
por: Piotter, Elena, et al.
Publicado: (2021)

The Scope of Pathogenic ABCA4 Mutations Targetable by CRISPR DNA Base Editing Systems—A Systematic Review
por: Piotter, Elena, et al.
Publicado: (2022)

CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin
por: Kaukonen, Maria, et al.
Publicado: (2022)

New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome
por: Major, Lauren, et al.
Publicado: (2022)

A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery
por: Major, Lauren, et al.
Publicado: (2023)

Accurate Quantification of AAV Vector Genomes by Quantitative PCR
por: Martinez-Fernandez de la Camara, Cristina, et al.
Publicado: (2021)

Minicircle Delivery to the Neural Retina as a Gene Therapy Approach
por: Staurenghi, Federica, et al.
Publicado: (2022)

Expression of Rab Prenylation Pathway Genes and Relation to Disease Progression in Choroideremia
por: Fry, Lewis E., et al.
Publicado: (2021)

Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances
por: McClements, Michelle E., et al.
Publicado: (2020)

Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases
por: Hansen, Silja, et al.
Publicado: (2023)

Function of human pluripotent stem cell-derived photoreceptor progenitors in blind mice
por: Barnea-Cramer, Alona O., et al.
Publicado: (2016)

CRISPR Interference–Potential Application in Retinal Disease
por: Peddle, Caroline F., et al.
Publicado: (2020)

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa
por: Fischer, M. Dominik, et al.
Publicado: (2017)

Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?
por: Bellingrath, Julia-Sophia, et al.
Publicado: (2023)

The Biological Activity of AAV Vectors for Choroideremia Gene Therapy Can Be Measured by In Vitro Prenylation of RAB6A
por: Patrício, Maria I., et al.
Publicado: (2018)

Molecular Strategies for RPGR Gene Therapy
por: Cehajic Kapetanovic, Jasmina, et al.
Publicado: (2019)

An analysis of the Kozak consensus in retinal genes and its relevance to gene therapy
por: McClements, Michelle E., et al.
Publicado: (2021)

 Gene Therapy for Color Blindness
por: Hassall, Mark M., et al.
Publicado: (2017)

Molecular Therapies for Choroideremia
por: Cehajic Kapetanovic, Jasmina, et al.
Publicado: (2019)

In Silico Analysis of Pathogenic CRB1 Single Nucleotide Variants and Their Amenability to Base Editing as a Potential Lead for Therapeutic Intervention
por: Bellingrath, Julia-Sophia, et al.
Publicado: (2021)

Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations
por: Salman, Ahmed, et al.
Publicado: (2022)

Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy
por: Patrício, Maria I., et al.
Publicado: (2019)

AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration
por: McClements, Michelle E., et al.
Publicado: (2021)

Impact of Vital Dyes on Cell Viability and Transduction Efficiency of AAV Vectors Used in Retinal Gene Therapy Surgery: An In Vitro and In Vivo Analysis
por: Salvetti, Anna P., et al.
Publicado: (2017)

Characterizing the Natural History of Visual Function in Choroideremia Using Microperimetry and Multimodal Retinal Imaging
por: Jolly, Jasleen K., et al.
Publicado: (2017)

AAV2/8 Anti-angiogenic Gene Therapy Using Single-Chain Antibodies Inhibits Murine Choroidal Neovascularization
por: Hughes, Chris P., et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_96vo600b2ol3o0h56mlrq9ncl0