Cargando…

Efficacy of dual intracerebroventricular and intravitreal CLN5 gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease

Mutations in the CLN5 gene cause the fatal, pediatric, neurodegenerative disease CLN5 neuronal ceroid lipofuscinosis. Affected children suffer progressive neuronal loss, visual failure and premature death. Presently there is no treatment. This study evaluated dual intracerebroventricular (ICV) and i...

Descripción completa

Detalles Bibliográficos
Autores principales:	Murray, Samantha J., Wellby, Martin P., Barrell, Graham K., Russell, Katharina N., Deane, Ashley R., Wynyard, John R., Gray, Steven J., Palmer, David N., Mitchell, Nadia L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2023
Materias:	Pharmacology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10628725/ https://www.ncbi.nlm.nih.gov/pubmed/37942487 http://dx.doi.org/10.3389/fphar.2023.1212235

Ejemplares similares

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)

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

Electroretinography data from ovine models of CLN5 and CLN6 neuronal ceroid lipofuscinoses
por: Russell, Katharina N., 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)

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)

Progressive MRI brain volume changes in ovine models of CLN5 and CLN6 neuronal ceroid lipofuscinosis
por: Murray, Samantha J, et al.
Publicado: (2023)

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

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

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

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

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

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)

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)

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)

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

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

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

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

Natural history of retinal degeneration in ovine models of CLN5 and CLN6 neuronal ceroid lipofuscinoses
por: Murray, S. J., et al.
Publicado: (2022)

Data on characterizing the gene expression patterns of neuronal ceroid lipofuscinosis genes: CLN1, CLN2, CLN3, CLN5 and their association to interneuron and neurotransmission markers: Parvalbumin and Somatostatin
por: Minye, Helena M., et al.
Publicado: (2016)

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

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

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)

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

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)

Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype
por: Soldati, Chiara, et al.
Publicado: (2021)

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)

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

Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration
por: Kim, Aryun, et al.
Publicado: (2020)

A comparative study of the degradation of yeast cyclins Cln1 and Cln2
por: Quilis, Inma, et al.
Publicado: (2016)

Loss of the batten disease protein CLN3 leads to mis-trafficking of M6PR and defective autophagic-lysosomal reformation
por: Calcagni’, Alessia, et al.
Publicado: (2023)

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)

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)

Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
por: White, Katherine A., et al.
Publicado: (2021)

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)

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep
por: Nelvagal, Hemanth R., et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_n4cve5p82cpu3m6uin1i1mi0nm