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المؤلفون: Ruozi, Giulia, Bortolotti, Francesca, Mura, Antonio, Tomczyk, Mateusz, Falcione, Antonella, Martinelli, Valentina, Vodret, Simone, Braga, Luca, Dal Ferro, Matteo, Cannatà, Antonio, Zentilin, Lorena, Sinagra, Gianfranco, Zacchigna, Serena, Giacca, Mauro
المساهمون: Ruozi, Giulia, Bortolotti, Francesca, Mura, Antonio, Tomczyk, Mateusz, Falcione, Antonella, Martinelli, Valentina, Vodret, Simone, Braga, Luca, Dal Ferro, Matteo, Cannatà, Antonio, Zentilin, Lorena, Sinagra, Gianfranco, Zacchigna, Serena, Giacca, Mauro
المصدر: Science Translational Medicine
مصطلحات موضوعية: Fibrosi, Myocardial Infarction, Nerve Tissue Proteins, Neoplasm Protein, Mice, Animals, Myocytes, Cardiac, Eye Proteins, Myofibroblasts, Cytokine, Secretome, Heart Failure, Myofibroblast, Ventricular Remodeling, Animal, Eye Protein, General Medicine, Dependovirus, Dependoviru, Fibrosis, Neoplasm Proteins, Mice, Inbred C57BL, Disease Models, Animal, Nerve Tissue Protein, Cytokines
وصف الملف: ELETTRONICO
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::305b61588730dc6b1f89f6d2e35c1f4b
https://pubmed.ncbi.nlm.nih.gov/36044596 -
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المؤلفون: Newman, Nancy J, Schniederjan, Matthew, Mendoza, Pia R, Calkins, David J, Yu-Wai-Man, Patrick, Biousse, Valérie, Carelli, Valerio, Taiel, Magali, Rugiero, Francois, Singh, Pramila, Rogue, Alexandra, Sahel, José-Alain, Ancian, Philippe
المساهمون: Newman, Nancy J [0000-0003-4898-7839], Apollo - University of Cambridge Repository, Newman N.J., Schniederjan M., Mendoza P.R., Calkins D.J., Yu-Wai-Man P., Biousse V., Carelli V., Taiel M., Rugiero F., Singh P., Rogue A., Sahel J.-A., Ancian P.
المصدر: BMC Neurology. 22
مصطلحات موضوعية: genetic structures, Biopsy, Recombinant adeno-associated virus vector 2 serotype 2, Optic Atrophy, Hereditary, Leber, General Medicine, Dependoviru, eye diseases, qPCR assay, Follow-Up Studie, Brain Neoplasm, Lenadogene nolparvovec, ND4, Clinical Trials, Phase III as Topic, Case report, Female, Viral vector transduction, Neurology (clinical), Glioblastoma, Leber hereditary optic neuropathy, Aged, Human
وصف الملف: text/xml; application/pdf; ELETTRONICO
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المؤلفون: Patrizia Tornabene, Rita Ferla, Manel Llado-Santaeularia, Miriam Centrulo, Margherita Dell’Anno, Federica Esposito, Elena Marrocco, Emanuela Pone, Renato Minopoli, Carolina Iodice, Edoardo Nusco, Settimio Rossi, Hristiana Lyubenova, Anna Manfredi, Lucio Di Filippo, Antonella Iuliano, Annalaura Torella, Giulio Piluso, Francesco Musacchia, Enrico Maria Surace, Davide Cacchiarelli, Vincenzo Nigro, Alberto Auricchio
المساهمون: Tornabene, Patrizia, Ferla, Rita, Llado-Santaeularia Miriam Centrulo, Manel, Dell'Anno, Margherita, Esposito, Federica, Marrocco, Elena, Pone, Emanuela, Minopoli, Renato, Iodice, Carolina, Nusco, Edoardo, Rossi, Settimio, Lyubenova, Hristiana, Manfredi, Anna, Di Filippo, Lucio, Iuliano, Antonella, Torella, Annalaura, Piluso, Giulio, Musacchia, Francesco, Maria Surace, Enrico, Cacchiarelli, Davide, Nigro, Vincenzo, Auricchio, Alberto, Tornabene, P., Ferla, R., Llado-Santaeularia, M., Centrulo, M., Dell'Anno, M., Esposito, F., Marrocco, E., Pone, E., Minopoli, R., Iodice, C., Nusco, E., Rossi, S., Lyubenova, H., Manfredi, A., Di Filippo, L., Iuliano, A., Torella, A., Piluso, G., Musacchia, F., Surace, E. M., Cacchiarelli, D., Nigro, V., Auricchio, A.
مصطلحات موضوعية: Gene Editing, Multidisciplinary, Animal, Swine, viruses, Genetic Vectors, General Physics and Astronomy, General Chemistry, Dependovirus, Dependoviru, General Biochemistry, Genetics and Molecular Biology, Retina, Mice, Liver, Animals, CRISPR-Cas System, Genetic Vector, CRISPR-Cas Systems
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bddefe9337552842eca9f4efdc9c2f5d
http://hdl.handle.net/11591/476671 -
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المؤلفون: Irena Tsui, Claudia B. Catarino, Piero Barboni, Günther Rudolph, Nancy J. Newman, Sara Silvestri, Alfredo A. Sadun, Michael Dattilo, Neringa Jurkute, Jean-François Girmens, Cosima Schertler, Magali Taiel, Rustum Karanjia, Claudia Priglinger, Maria K Gemenetzi, Armin Wolf, Manuela Contin, Jasmina Al-Tamami, Thomas Klopstock, James Acheson, Robert C. Sergott, Deborah Gibbs, Rabih Hage, Stephan R. Thurau, Adam A. DeBusk, Lidia Di Vito, Mark L. Moster, Valérie Biousse, Med Lindreth DuBois, Valerio Carelli, Gad Heilweil, Chiara La Morgia, Michele Carbonelli, Andrew Hendrick, Patrick Yu-Wai-Man, Jason H. Peragallo, Gerard Smits, Angelika Pressler, Martin Hildebrandt, Alcides Fernandes Filho, Michael Neuenhahn, Bettina von Livonius, Barrett Katz, Daniel R Muth, Siegfried G. Priglinger, Lauren Leitch-Devlin, Susan Mohamed, William R. Tucker, Maria Massini, Maria Eleftheriadou, Laure Blouin, Catherine Vignal-Clermont, Eman Hawy, Simona Degli Esposti, Heather Tollis, G. Baker Hubbard, Jannah Rutter Dobbs, José-Alain Sahel, Catherine Vignal, Melissa SantaMaria, Julie A. Haller
المساهمون: Emory University School of Medicine, Emory University [Atlanta, GA], Addenbrooke's Hospital, Cambridge University NHS Trust, Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS), University of Bologna, Jefferson (Philadelphia University + Thomas Jefferson University), Fondation Ophtalmologique Adolphe de Rothschild [Paris], Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (CHNO), Ludwig-Maximilians-Universität München (LMU), University of California [Los Angeles] (UCLA), University of California, Ludwig Maximilian University [Munich] (LMU), Institut de la Vision, Centre National de la Recherche Scientifique (CNRS)-Sorbonne Université (SU)-Institut National de la Santé et de la Recherche Médicale (INSERM), University of Pittsburgh School of Medicine, Pennsylvania Commonwealth System of Higher Education (PCSHE), Institut Hospitalo-Universitaire FOReSIGHT, Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (CHNO)-Sorbonne Université (SU), Newman, NJ, Yu-Wai-Man, P, Carelli, V, Moster, ML, Biousse, V, Vignal-Clermont, C, Sergott, RC, Klopstock, T, Sadun, AA, Barboni, P, DeBusk, AA, Girmens, JF, Rudolph, G, Karanjia, R, Taiel, M, Blouin, L, Smits, G, Katz, B, Sahel, J-A, Vignal, C, Hage, R, Catarino, CB, Priglinger, C, Priglinger, S, Thurau, S, von Livonius, B, Muth, D, Wolf, A, Al-Tamami, J, Pressler, A, Schertler, C, Hildebrandt, M, Neuenhahn, M, Heilweil, G, Tsui, I, Hubbard, GB, Hendrick, A, Dattilo, M, Peragallo, J, Hawy, E, DuBois, Med L, Gibbs, D, Filho, AF, Dobbs, J, Carbonelli, M, Di Vito, L, Contin, M, Mohamed, S, La Morgia, C, Silvestri, S, Acheson, J, Eleftheriadou, M, Esposti, S, Gemenetzi, M, Leitch-Devlin, L, Tucker, WR, Jurkute, N, SantaMaria, M, Tollis, H, Haller, JA, Massini, M.
المصدر: Ophthalmology: Journal of The American Academy of Ophthalmology
Ophthalmology: Journal of The American Academy of Ophthalmology, Elsevier, 2021, 128 (5), pp.649-660. ⟨10.1016/j.ophtha.2020.12.012⟩
Ophthalmology 128(5), 649-660 (2021). doi:10.1016/j.ophtha.2020.12.012مصطلحات موضوعية: Male, Time Factors, Visual acuity, genetic structures, physiology [Visual Fields], [SDV]Life Sciences [q-bio], efficacy, Visual Acuity, Phases of clinical research, genetics [Dependovirus], chemistry.chemical_compound, 0302 clinical medicine, Visual Field Test, Quality of life, Clinical endpoint, Contrast (vision), intravitreal gene therapy, media_common, 0303 health sciences, physiology [Visual Acuity], Diabetic retinopathy, Dependovirus, Middle Aged, Dependoviru, genetics [DNA, Mitochondrial], Phase 3 randomized double-masked clinical trial, Treatment Outcome, Intravitreal Injections, diagnosis [Optic Atrophy, Hereditary, Leber], Female, Genetic Vector, medicine.symptom, Human, Adult, safety, LEBER HEREDITARY OPTIC NEUROPATHY, medicine.medical_specialty, retinal anatomic measures, Time Factor, Adolescent, media_common.quotation_subject, psychology [Optic Atrophy, Hereditary, Leber], Genetic Vectors, Visual Field, Humphrey visual field perimetry, Optic Atrophy, Hereditary, Leber, therapy [Optic Atrophy, Hereditary, Leber], DNA, Mitochondrial, bilateral visual improvement, Follow-Up Studie, Leber Hereditary Optic Neuropathy, Young Adult, 03 medical and health sciences, Double-Blind Method, Ophthalmology, psychology [Quality of Life], Electroretinography, medicine, Humans, ddc:610, Aged, 030304 developmental biology, contrast sensitivity, business.industry, Intravitreal Injection, Retinal, Genetic Therapy, retinal anatomic measure, medicine.disease, eye diseases, genetics [Optic Atrophy, Hereditary, Leber], chemistry, Mutation, 030221 ophthalmology & optometry, Visual Field Tests, sense organs, Visual Fields, business, Follow-Up Studies, best-corrected visual acuity
وصف الملف: STAMPA
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المؤلفون: Alberto Auricchio, Enrico Maria Surace, Clarissa Patrizi, Michael E. Cheetham, Daniela Benati, Manel Llado, Alessandra Recchia, Rosellina Guarascio, Carolina Iodice, Elena Marrocco
المساهمون: Patrizi, C., Llado, M., Benati, D., Iodice, C., Marrocco, E., Guarascio, R., Surace, E. M., Cheetham, M. E., Auricchio, A., Recchia, A.
المصدر: American Journal of Human Genetics
مصطلحات موضوعية: 0301 basic medicine, Transgenic, Mice, 0302 clinical medicine, Genome editing, INDEL Mutation, CRISPR, Missense mutation, CRISPR-Cas System, Genetics (clinical), Genetics, Allele, Gene Editing, biology, CRISPR-Cas9 editing, Dependovirus, Dependoviru, AAV vector, Rhodopsin, retinitis pigmentosa, transgenic mice, Alleles, Animals, CRISPR-Cas Systems, Cell Line, Disease Models, Animal, Electroretinography, Genetic Therapy, Humans, Mice, Transgenic, Mutation, Missense, Photoreceptor Cells, Vertebrate, Retina, Retinitis Pigmentosa, Human, Genetically modified mouse, Article, 03 medical and health sciences, Retinitis pigmentosa, medicine, Photoreceptor Cells, Gene, Vertebrate, Animal, medicine.disease, eye diseases, 030104 developmental biology, Mutation, Disease Models, 030221 ophthalmology & optometry, biology.protein, Missense
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6d8264c39b9375f77bccebc666a992f6
http://hdl.handle.net/11588/854858 -
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المؤلفون: Charlotte Demkes, Mauro Giacca, Bastiaan Spanjaard, Farhad Akbari Moqadam, Lieneke Kooijman, Hesther de Ruiter, Lorena Zentilin, D Versteeg, Bas Molenaar, Anne Katrine Johansen, Eva van Rooij, Ana Rita Leitoguinho
المساهمون: Johansen, Anne Katrine, Molenaar, Ba, Versteeg, Danielle, Leitoguinho, Ana Rita, Demkes, Charlotte, Spanjaard, Bastiaan, de Ruiter, Hesther, Akbari Moqadam, Farhad, Kooijman, Lieneke, Zentilin, Lorena, Giacca, Mauro, van Rooij, Eva, Hubrecht Institute for Developmental Biology and Stem Cell Research
المصدر: Circulation Research, 121(10), 1168-1181. Lippincott Williams & Wilkins
مصطلحات موضوعية: 0301 basic medicine, sequence analysis, Physiology, Inbred C57BL, Genome, Transgenic, chemistry.chemical_compound, Mice, Genome editing, molecular biology, RNA, Guide, CRISPR, CRISPR-Cas System, Myocytes, Cardiac, Guide RNA, NIH 3T3 Cell, Cells, Cultured, Mice, Knockout, Gene Editing, Cultured, Palindrome, Gene Transfer Techniques, Dependovirus, Dependoviru, clustered regularly interspaced short palindromic repeats, gene editing, myocytes, cardiac, sequence analysis, DNA, Animals, Animals, Newborn, Base Sequence, CRISPR-Cas Systems, Mice, Inbred C57BL, Mice, Transgenic, NIH 3T3 Cells, Cardiology and Cardiovascular Medicine, Cardiac, RNA, Guide, Kinetoplastida, Cells, Knockout, Computational biology, Biology, Article, 03 medical and health sciences, Journal Article, Gene, Myocytes, Animal, Cas9, DNA, Gene Transfer Technique, Newborn, 030104 developmental biology, chemistry, RNA, clustered regularly interspaced short palindromic repeat, Guide
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bb66c30d26a595eda1fb2d3c95d584e3
https://doi.org/10.1161/circresaha.116.310370 -
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المؤلفون: Giancarlo Parenti, Karen Kozarsky, John J. Hopwood, Rita Ferla, Generoso Andria, Alberto Auricchio, Ans T. van der Ploeg, Rossella Parini, Marco Savarese, Maurizio Scarpa, Hatice Serap Sivri, Vincenzo Nigro, Pamela Claudiani, Nicola Brunetti-Pierri, Maria Alice Donati, Giovanni Sorge, Simona Fecarotta
المساهمون: Ferla, R, Claudiani, P, Savarese, M, Kozarsky, K., Parini, R, Scarp, a M, Donati, Ma, Sorge, G, Hopwood, Jj, Parenti, Giancarlo, Fecarotta, S, Nigro, V, Sivri, H, Van Der Ploeg, A, Andria, Generoso, BRUNETTI PIERRI, Nicola, Auricchio, Alberto, Çocuk Sağlığı ve Hastalıkları, Ferla, Rita, Claudiani, Pamela, Savarese, Marco, Kozarsky, Karen, Parini, Rossella, Scarpa, Maurizio, Donati, Maria Alice, Sorge, Giovanni, Hopwood, John J., Fecarotta, Simona, Nigro, Vincenzo, Sivri, Hatice Serap, Van Der Ploeg, An, Brunetti Pierri, Nicola, Pediatrics
المصدر: Human Gene Therapy
Human Gene Therapy, 26(3), 145-152. Mary Ann Liebert Inc.مصطلحات موضوعية: Arylsulfatase B, Turkey, N-Acetylgalactosamine-4-Sulfatase, Genetic enhancement, DNA Mutational Analysis, medicine.disease_cause, Cohort Studies, 0302 clinical medicine, Medicine, Neutralizing, Adeno-associated virus, Research Articles, Netherlands, 0303 health sciences, Mucopolysaccharidosis VI, biology, Medicine (all), Dependovirus, Dependoviru, 3. Good health, Italy, 030220 oncology & carcinogenesis, Cross Reaction, Molecular Medicine, Antibody, Human, Cross Reactions, Antibodies, Neutralizing, Genetic Therapy, Humans, Mutation, Patient Selection, Molecular Biology, Genetics, Antibodies, Virus, Frameshift mutation, DNA Mutational Analysi, 03 medical and health sciences, Netherland, 030304 developmental biology, business.industry, Virology, Immunology, biology.protein, Cohort Studie, business
وصف الملف: text/plain
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::43f387a870106574d90a0027e2392059
http://europepmc.org/articles/PMC4367235 -
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المؤلفون: Lorena Zentilin, Francesca Bortolotti, Sara Doimo, Lawrence Banks, Antonella Falcione, Pierluigi Lesizza, Mauro Giacca, Giulia Ruozi, Matteo Dal Ferro, Serena Zacchigna
المساهمون: Bortolotti, Francesca, Ruozi, Giulia, Falcione, Antonella, Doimo, Sara, Dal Ferro, Matteo, Lesizza, Pierluigi, Zentilin, Lorena, Banks, Lawrence, Zacchigna, Serena, Giacca, Mauro
المصدر: Circulation. 136(16)
مصطلحات موضوعية: 0301 basic medicine, Male, Time Factors, cardiotrophin-1, cell- and tissue-based therapy, focal adhesions, genetic vectors, mesenchymal stromal cells, myocardial infarction, Animals, Apoptosis, Cell Adhesion, Cell Proliferation, Cell Survival, Cells, Cultured, Cytokines, Dependovirus, Disease Models, Animal, Focal Adhesion Kinase 1, Gene Library, Genetic Vectors, Graft Survival, Mesenchymal Stromal Cells, Mice, Inbred C57BL, Myocardial Contraction, Myocardial Infarction, Myocardium, Recovery of Function, STAT3 Transcription Factor, Signal Transduction, Transduction, Genetic, Transfection, Mesenchymal Stem Cell Transplantation, Regeneration, Cardiology and Cardiovascular Medicine, Physiology (medical), Cell, 030204 cardiovascular system & hematology, Inbred C57BL, Mice, 0302 clinical medicine, Myocardial infarction, focal adhesion, Cultured, Mesenchymal Stromal Cell, Dependoviru, genetic vector, medicine.anatomical_structure, Time Factor, Cardiotrophin 1, Cells, Transduction, 03 medical and health sciences, Genetic, In vivo, medicine, Cytokine, Animal, business.industry, Regeneration (biology), Mesenchymal stem cell, Apoptosi, Mesenchymal Stem Cells, medicine.disease, Transplantation, 030104 developmental biology, Disease Models, Immunology, Cancer research, business
وصف الملف: STAMPA
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::131c13fd761379973e533d13e30af79f
https://pubmed.ncbi.nlm.nih.gov/29038207 -
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المؤلفون: Jérôme Ausseil, Béatrice Husson, Dimitrios I. Zafeiriou, Kumaran Deiva, Valérie Furlan, Béatrice Poirier, Christian Meyer, Philippe Bourget, Stéphanie de Bournonville, Cecile Artaud, Marc Tardieu, Thomas Roujeau, Jean-Michel Heard, Giancarlo Parenti, Thomas Baugnon, Marie-Lise Gougeon, M. Zerah, Ronald G. Crystal
المساهمون: Tardieu, Marc, Zérah, Michel, Gougeon, Marie lise, Ausseil, Jérome, De Bournonville, Stéphanie, Husson, Béatrice, Zafeiriou, Dimitrio, Parenti, Giancarlo, Bourget, Philippe, Poirier, Béatrice, Furlan, Valérie, Artaud, Cécile, Baugnon, Thoma, Roujeau, Thoma, Crystal, Ronald G, Meyer, Christian, Deiva, Kumaran, Heard, Jean michel
المصدر: The Lancet. Neurology. 16(9)
مصطلحات موضوعية: 0301 basic medicine, Pediatrics, medicine.medical_specialty, Genetic Vectors, Disease, Mucopolysaccharidosis type III, Immunosuppressive Agent, Outcome Assessment (Health Care), 03 medical and health sciences, Mucopolysaccharidosis III, Immune system, Acetylglucosaminidase, Outcome Assessment, Health Care, Lysosomal storage disease, medicine, Humans, Adverse effect, business.industry, Brain, Infant, Genetic Therapy, Syndrome, Dependovirus, Dependoviru, medicine.disease, Natural history, Clinical trial, 030104 developmental biology, Child, Preschool, Genetic Vector, Neurology (clinical), business, Neurocognitive, Immunosuppressive Agents, Human
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المؤلفون: Alberto Auricchio, Katherine A. High, Julie DiStefano-Pappas, Kathleen A. Marshall, Dominique Cross, J. Fraser Wright, Albert M. Maguire, Puya Aravand, Okan U. Elci, Sarah McCague, Federico Mingozzi, Laura Cyckowski, Bart P. Leroy, Daniel C. Chung, Jennifer Wellman, Jason Ruggiero, Francesca Simonelli, Eric A. Pierce, Junwei Sun, Manzar Ashtari, Jeannette L. Bennicelli, Jean Bennett
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