-
1دورية أكاديمية
المؤلفون: Tyler B. Johnson, Jon J. Brudvig, Shibi Likhite, Melissa A. Pratt, Katherine A. White, Jacob T. Cain, Clarissa D. Booth, Derek J. Timm, Samantha S. Davis, Brandon Meyerink, Ricardo Pineda, Cassandra Dennys-Rivers, Brian K. Kaspar, Kathrin Meyer, Jill M. Weimer
المصدر: Frontiers in Genetics, Vol 14 (2023)
مصطلحات موضوعية: neuronal ceroid lipofuscinosis, neurodegenerative disease, gene therapy, adeno-associated virus, rare disease, Genetics, QH426-470
وصف الملف: electronic resource
-
2
المؤلفون: Brandon Meyerink, Kathrin Meyer, Logan Langin, Derek J. Timm, Jacob T. Cain, Tyler B. Johnson, Katherine A. White, Samantha Davis, Clarissa D. Booth, Melissa A. Pratt, Jill M. Weimer, Shibi Likhite, Jon Brudvig
مصطلحات موضوعية: 0303 health sciences, Batten disease, business.industry, Transgene, Genetic enhancement, Disease, medicine.disease, Bioinformatics, 3. Good health, 03 medical and health sciences, 0302 clinical medicine, CLN8, medicine, Neuronal ceroid lipofuscinosis, medicine.symptom, business, Pathological, Myoclonus, 030217 neurology & neurosurgery, 030304 developmental biology
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2afbfed0ada49ecbd41a56da575b3fde
https://doi.org/10.1101/2020.05.05.079350 -
3
المؤلفون: Brandon Meyerink, Derek J. Timm, Melissa A. Pratt, Kathrin Meyer, Jacob T. Cain, Jill M. Weimer, Katherine A. White, Shibi Likhite, Samantha Davis, Tyler B. Johnson, Logan Langin, Clarissa D. Booth, Jon Brudvig
المصدر: Mol Ther
مصطلحات موضوعية: Batten disease, Genetic enhancement, Transgene, Genetic Vectors, Gene Expression, Disease, Bioinformatics, 03 medical and health sciences, Mice, 0302 clinical medicine, Neuronal Ceroid-Lipofuscinoses, Drug Discovery, Genetics, medicine, Animals, Humans, Transgenes, Molecular Biology, 030304 developmental biology, Pharmacology, 0303 health sciences, Behavior, Animal, business.industry, Neurodegeneration, Membrane Proteins, Genetic Therapy, Dependovirus, medicine.disease, Disease Models, Animal, Treatment Outcome, CLN8, 030220 oncology & carcinogenesis, Molecular Medicine, Neuronal ceroid lipofuscinosis, Original Article, medicine.symptom, business, Myoclonus
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b7b85864e17561c9a742537fa6bd48b5
https://pubmed.ncbi.nlm.nih.gov/33010819 -
4
المؤلفون: Alberto di Ronza, Pengcheng Zhang, Lakshya Bajaj, Dany Roman, Rituraj Pal, Sung Yun Jung, Jill M. Weimer, Jaiprakash Sharma, Rui Chen, Marco Sardiello, Richard N. Sifers, Randy Schekman, John R. Collette, Aiden Eblimit, Clarissa D. Booth, Kevin T. Chang
المصدر: J Clin Invest
مصطلحات موضوعية: 0301 basic medicine, Batten disease, Golgi Apparatus, Endoplasmic Reticulum, 03 medical and health sciences, symbols.namesake, Mice, 0302 clinical medicine, Neuronal Ceroid-Lipofuscinoses, Lysosome, medicine, Animals, chemistry.chemical_classification, Mice, Knockout, Endoplasmic reticulum, Mutagenesis, Membrane Proteins, General Medicine, Golgi apparatus, medicine.disease, Cell biology, Protein Transport, 030104 developmental biology, Enzyme, medicine.anatomical_structure, chemistry, CLN8, 030220 oncology & carcinogenesis, Multiprotein Complexes, symbols, Lysosomes, Biogenesis, Research Article