Cargando…

Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice

Batten disease is a family of rare, fatal, neuropediatric diseases presenting with memory/learning decline, blindness, and loss of motor function. Recently, we reported the use of an AAV9-mediated gene therapy that prevents disease progression in a mouse model of CLN6-Batten disease (Cln6(nclf)), re...

Descripción completa

Detalles Bibliográficos
Autores principales:	White, Katherine A., Nelvagal, Hemanth R., Poole, Timothy A., Lu, Bin, Johnson, Tyler B., Davis, Samantha, Pratt, Melissa A., Brudvig, Jon, Assis, Ana B., Likhite, Shibi, Meyer, Kathrin, Kaspar, Brian K., Cooper, Jonathan D., Wang, Shaomei, Weimer, Jill M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7887332/ https://www.ncbi.nlm.nih.gov/pubmed/33665223 http://dx.doi.org/10.1016/j.omtm.2020.12.014

Ejemplares similares

Sex-split analysis of pathology and motor-behavioral outcomes in a mouse model of CLN8-Batten disease reveals an increased disease burden and trajectory in female Cln8(mnd) mice
por: Holmes, Andrew D., et al.
Publicado: (2022)

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease
por: Cain, Jacob T., et al.
Publicado: (2019)

Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease
por: Johnson, Tyler B., et al.
Publicado: (2023)

A novel porcine model of CLN3 Batten disease recapitulates clinical phenotypes
por: Swier, Vicki J., et al.
Publicado: (2023)

A tailored Cln3(Q352X) mouse model for testing therapeutic interventions in CLN3 Batten disease
por: Langin, Logan, et al.
Publicado: (2020)

Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach
por: Centa, Jessica L., et al.
Publicado: (2023)

Tracking sex-dependent differences in a mouse model of CLN6-Batten disease
por: Poppens, McKayla J., et al.
Publicado: (2019)

Searching for novel biomarkers using a mouse model of CLN3-Batten disease
por: Timm, Derek, et al.
Publicado: (2018)

Magnetic resonance brain volumetry biomarkers of CLN2 Batten disease identified with miniswine model
por: Knoernschild, Kevin, et al.
Publicado: (2023)

CLN7 gene therapy: hope for an ultra-rare condition
por: Brudvig, Jon J., et al.
Publicado: (2022)

A human model of Batten disease shows role of CLN3 in phagocytosis at the photoreceptor–RPE interface
por: Tang, Cynthia, et al.
Publicado: (2021)

Glycerophosphoinositol is Elevated in Blood Samples From CLN3(Δex7-8) pigs, Cln3(Δex7-8) Mice, and CLN3-Affected Individuals
por: Brudvig, Jon J, et al.
Publicado: (2022)

Molecular neuropathology of the synapse in sheep with CLN5 Batten disease
por: Amorim, Inês S., et al.
Publicado: (2015)

Modulation of CRMP2 via (S)-Lacosamide shows therapeutic promise but is ultimately ineffective in a mouse model of CLN6-Batten disease
por: White, Katherine A., et al.
Publicado: (2019)

The Batten disease gene CLN3 is required for the response to oxidative stress
por: Tuxworth, Richard I., et al.
Publicado: (2011)

Efficacy of phosphodiesterase‐4 inhibitors in juvenile Batten disease (CLN3)
por: Aldrich, Amy, et al.
Publicado: (2016)

An ERG and OCT study of neuronal ceroid lipofuscinosis CLN2 Battens retinopathy
por: Thompson, Dorothy A., et al.
Publicado: (2021)

Neurodegeneration and Epilepsy in a Zebrafish Model of CLN3 Disease (Batten Disease)
por: Wager, Kim, et al.
Publicado: (2016)

Discovery of a CLN7 model of Batten disease in non-human primates
por: McBride, Jodi L., et al.
Publicado: (2018)

Recognizing differentiating clinical signs of CLN3 disease (Batten disease) at presentation
por: Kuper, Willemijn F. E., et al.
Publicado: (2020)

Correction: An ERG and OCT study of neuronal ceroid lipofuscinosis CLN2 Battens retinopathy
por: Thompson, Dorothy A., et al.
Publicado: (2021)

Osmotic Stress Changes the Expression and Subcellular Localization of the Batten Disease Protein CLN3
por: Getty, Amanda, et al.
Publicado: (2013)

Interactions between the juvenile Batten disease gene, CLN3, and the Notch and JNK signalling pathways
por: Tuxworth, Richard I., et al.
Publicado: (2009)

Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics
por: Rowe, Ashley A., et al.
Publicado: (2022)

The Batten disease protein CLN3 is important for stress granules dynamics and translational activity
por: Relton, Emily L., et al.
Publicado: (2023)

Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease
por: Mitchell, Nadia L., et al.
Publicado: (2023)

Efficacy of dual intracerebroventricular and intravitreal CLN5 gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease
por: Murray, Samantha J., et al.
Publicado: (2023)

Automatic quantification of lymphocyte vacuolization in peripheral blood smears of patients with Batten's disease (CLN3 disease)
por: Nonkes, Lourens J. P., et al.
Publicado: (2021)

Translational neurophysiology in sheep: measuring sleep and neurological dysfunction in CLN5 Batten disease affected sheep
por: Perentos, Nicholas, et al.
Publicado: (2015)

Transmembrane Batten Disease Proteins Interact With a Shared Network of Vesicle Sorting Proteins, Impacting Their Synaptic Enrichment
por: Rechtzigel, Mitchell J., et al.
Publicado: (2022)

Lack of specificity of antibodies raised against CLN3, the lysosomal/endosomal transmembrane protein mutated in juvenile Batten disease
por: Nelson, Tarah, et al.
Publicado: (2017)

In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans
por: Schwartz, Maura K., et al.
Publicado: (2023)

Juvenile Batten Disease (CLN3): Detailed Ocular Phenotype, Novel Observations, Delayed Diagnosis, Masquerades, and Prospects for Therapy
por: Wright, Genevieve A., et al.
Publicado: (2020)

Finding the most appropriate mouse model of juvenile CLN3 (Batten) disease for therapeutic studies: the importance of genetic background and gender
por: Kovács, Attila D., et al.
Publicado: (2015)

Comparative proteomic profiling reveals mechanisms for early spinal cord vulnerability in CLN1 disease
por: Nelvagal, Hemanth R., et al.
Publicado: (2020)

Aggregation chimeras provide evidence of in vivo intercellular correction in ovine CLN6 neuronal ceroid lipofuscinosis (Batten disease)
por: Barry, Lucy Anne, et al.
Publicado: (2022)

Towards Understanding Behaviour and Emotions of Children with CLN3 Disease (Batten Disease): Patterns, Problems and Support for Child and Family
por: Honingh, Aline K., et al.
Publicado: (2022)

Rapid and Progressive Regional Brain Atrophy in CLN6 Batten Disease Affected Sheep Measured with Longitudinal Magnetic Resonance Imaging
por: Sawiak, Stephen J., et al.
Publicado: (2015)

Spectrum of Ocular Manifestations in CLN2-Associated Batten (Jansky-Bielschowsky) Disease Correlate with Advancing Age and Deteriorating Neurological Function
por: Orlin, Anton, et al.
Publicado: (2013)

Extracellular Vesicles as Drug Carriers for Enzyme Replacement Therapy to Treat CLN2 Batten Disease: Optimization of Drug Administration Routes
por: Haney, Matthew J., et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_ju9qrncnnav5modi529icnukn8