Found: 19
Select item for more details and to access through your institution.
Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease).
- Published in:
- Scientific Reports, 2018, v. 8, n. 1, p. 1, doi. 10.1038/s41598-018-33449-0
- By:
- Publication type:
- Article
Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway.
- Published in:
- Human Molecular Genetics, 2014, v. 23, n. 8, p. 2005, doi. 10.1093/hmg/ddt596
- By:
- Publication type:
- Article
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.
- Published in:
- Science Translational Medicine, 2020, v. 12, n. 572, p. 1, doi. 10.1126/scitranslmed.abb5413
- By:
- Publication type:
- Article
Gene therapy for metachromatic leukodystrophy.
- Published in:
- Journal of Neuroscience Research, 2016, v. 94, n. 11, p. 1169, doi. 10.1002/jnr.23792
- By:
- Publication type:
- Article
Novel Therapies, High-Risk Pediatric Research, and the Prospect of Benefit: Learning from the Ethical Disagreements.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 6, p. 1095, doi. 10.1038/mt.2012.90
- By:
- Publication type:
- Article
Enhanced Survival of the LINCL Mouse Following CLN2 Gene Transfer Using the rh.10 Rhesus Macaque-derived Adeno-associated Virus Vector.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 3, p. 481, doi. 10.1038/sj.mt.6300049
- By:
- Publication type:
- Article
890. Improvement of Behavior and Mortality Following CNS Administration of AAVrh.10hCLN2 to CLN2 −/− Mice, a Model LINCL (Battan Disease) a Fatal Childhood Neurological Disorder (Batten Disease).
- Published in:
- Molecular Therapy, 2006, v. 13, p. S343, doi. 10.1016/j.ymthe.2006.08.979
- By:
- Publication type:
- Article
315. Identification and Control of Bacterial Contamination in an Academic Good Manufacturing Practice Facility.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S120, doi. 10.1016/j.ymthe.2006.08.371
- By:
- Publication type:
- Article
969. Development of Non-Invasive Imaging Strategies to Assess the Impact of Gene Therapy: Phenotypic Assessment of the Neurodegenerative CNS Disorder Late Infantile Neuronal Ceroid Lipofuscinosis Using Magnetic Resonance Spectroscopy
- Published in:
- 2005
- By:
- Publication type:
- Abstract
660. Quantitative Comparison of AAV Serotypes AAV2, AAV5, and AAVrh.10 Efficiency for CNS Gene Therapy Following Intracranial Gene Delivery
- Published in:
- 2005
- By:
- Publication type:
- Abstract
427. AAV2- and AAV5-Mediated CNS Delivery of Human CLN2 Reduces Lysosomal Storage in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis
- Published in:
- 2005
- By:
- Publication type:
- Abstract
Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice.
- Published in:
- Journal of Neuroscience, 2016, v. 36, n. 49, p. 12425, doi. 10.1523/JNEUROSCI.2016-16.2016
- By:
- Publication type:
- Article
Spectrum of Ocular Manifestations in CLN2-Associated Batten (Jansky-Bielschowsky) Disease Correlate with Advancing Age and Deteriorating Neurological Function.
- Published in:
- PLoS ONE, 2013, v. 8, n. 8, p. 1, doi. 10.1371/journal.pone.0073128
- By:
- Publication type:
- Article
Cocaine vaccine dAd5GNE protects against moderate daily and high-dose "binge" cocaine use.
- Published in:
- PLoS ONE, 2020, v. 15, n. 11, p. 1, doi. 10.1371/journal.pone.0239780
- By:
- Publication type:
- Article
Long‐term functional correction of cystathionine β‐synthase deficiency in mice by adeno‐associated viral gene therapy.
- Published in:
- Journal of Inherited Metabolic Disease, 2021, v. 44, n. 6, p. 1382, doi. 10.1002/jimd.12437
- By:
- Publication type:
- Article
Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma.
- Published in:
- PLoS ONE, 2016, v. 11, n. 10, p. 1, doi. 10.1371/journal.pone.0162978
- By:
- Publication type:
- Article
Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis.
- Published in:
- Journal of Neuroscience, 2006, v. 26, n. 5, p. 1334, doi. 10.1523/JNEUROSCI.2676-05.2006
- By:
- Publication type:
- Article
Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema.
- Published in:
- Allergy, 2019, v. 74, n. 6, p. 1081, doi. 10.1111/all.13582
- By:
- Publication type:
- Article
Expression and processing of mature human frataxin after gene therapy in mice.
- Published in:
- Scientific Reports, 2024, v. 14, n. 1, p. 1, doi. 10.1038/s41598-024-59060-0
- By:
- Publication type:
- Article