نتائج البحث - "Laura Cyckowski"

المساهمون: 73 M., Ashtari, L. L., Cyckowski, J. F., Monroe, K. A., Marshall, D. C., Chung, Auricchio, Alberto, F., Simonelli, B. P., Leroy, A. M., Meguire, K. S., Shindler, J., Bennett

المصدر: JOURNAL OF CLINICAL INVESTIGATION

مصطلحات موضوعية: Pathology, genetic structures, Genetic enhancement, Leber Congenital Amaurosis, Visual system, CORTICAL MAPS, Medicine and Health Sciences, Medicine, Dog Diseases, ROD, Visual Cortex, Clinical Trials, Phase I as Topic, Retinal Degeneration, General Medicine, CHILDHOOD BLINDNESS, RPE65 MUTATIONS, Visual field, Protein Transport, medicine.anatomical_structure, Technical Advance, Corrigendum, Microtubule-Associated Proteins, REORGANIZATION, cis-trans-Isomerases, medicine.medical_specialty, LEBER CONGENITAL AMAUROSIS, Genes, Recessive, Genetic Heterogeneity, Dogs, Animals, Humans, Sensory deprivation, Eye Proteins, Photoreceptor Connecting Cilium, RESTORATION, business.industry, Genetic Therapy, Recovery of Function, Macular degeneration, MACULAR DEGENERATION, medicine.disease, eye diseases, CANINE MODEL, Disease Models, Animal, VISION, Visual cortex, RPE65, Cis-trans-Isomerases, Sensory Deprivation, Carrier Proteins, business, Forecasting

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::82fecb0b6496f684fb214e28dd9921ae
https://doi.org/10.1172/jci57377

View record in OpenAIRE

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Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

المساهمون: Bennett, Jean, Wellman, Jennifer, Marshall, Kathleen A, Mccague, Sarah, Ashtari, Manzar, DiStefano Pappas, Julie, Elci, Okan U, Chung, Daniel C, Sun, Junwei, Wright, J. Fraser, Cross, Dominique R, Aravand, Puya, Cyckowski, Laura L, Bennicelli, Jeannette L, Mingozzi, Federico, Auricchio, Alberto, Pierce, Eric A, Ruggiero, Jason, Leroy, Bart P, Simonelli, Francesca, High, Katherine A, Maguire, Albert M., Institute of Geophysics, University of Tehran, Technologies et systèmes d'information pour les agrosystèmes (UR TSCF), Institut national de recherche en sciences et technologies pour l'environnement et l'agriculture (IRSTEA), Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE), École pratique des hautes études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Généthon, Laboratoire Aimé Cotton (LAC), Centre National de la Recherche Scientifique (CNRS)-Université Paris-Sud - Paris 11 (UP11)-École normale supérieure - Cachan (ENS Cachan), École Pratique des Hautes Études (EPHE), École normale supérieure - Cachan (ENS Cachan)-Université Paris-Sud - Paris 11 (UP11)-Centre National de la Recherche Scientifique (CNRS)

المصدر: LANCET
LANCET, 2016, 388 (10045), pp.661-72. ⟨10.1016/S0140-6736(16)30371-3⟩
The Lancet
The Lancet, 2016, 388 (10045), pp.661-72. ⟨10.1016/S0140-6736(16)30371-3⟩

مصطلحات موضوعية: 0301 basic medicine, Male, Retinal Rod Photoreceptor Cell, Genetic enhancement, [SDV]Life Sciences [q-bio], Administration, Ophthalmic, Disease, Blindness, chemistry.chemical_compound, Retinal Rod Photoreceptor Cells, Medicine, Age of Onset, Child, Evidence-Based Medicine, General Medicine, Dependovirus, Middle Aged, Dependoviru, 3. Good health, cis-trans-Isomerase, Blindne, Retreatment, Retinal Cone Photoreceptor Cells, Linear Model, Female, Genetic Vector, Occipital Lobe, Patient Safety, Injections, Intraocular, Retinal Cone Photoreceptor Cell, Human, cis-trans-Isomerases, Adult, medicine.medical_specialty, Adolescent, Genetic Vectors, Follow-Up Studie, 03 medical and health sciences, Cataracts, Ophthalmology, Humans, Adverse effect, Vision, Ocular, business.industry, Retinal, Genetic Therapy, medicine.disease, Clinical trial, 030104 developmental biology, chemistry, Cis-trans-Isomerases, Mutation, Linear Models, Age of onset, business, Follow-Up Studies