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Pathological modeling of glycogen storage disease type III with CRISPR/Cas9 edited human pluripotent stem cells.

학술저널

issue: Frontiers in cell and developmental biology [Front Cell Dev Biol] 2023 May 11; Vol. 11, pp. 1163427. Date of Electronic Publication: 2023 May 11 (Print Publication: 2023).
Author: Rossiaud L; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.
Fragner P; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.
Barbon E; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.
Gardin A; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.
Benabides M; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.
Pellier E; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.
Cosette J; Genethon, Evry, France.
El Kassar L; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.
Giraud-Triboult K; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.
Nissan X; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.
Ronzitti G; Genethon, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.
Hoch L; CECS, I-Stem, Corbeil-Essonnes, France.; INSERM U861, I-Stem, Corbeil-Essonnes, France.; UEVE U861, I-Stem, Corbeil-Essonnes, France.
DB Label: Database : MEDLINE

원문보기: Open Access (PubMed Central) 등재 - Web of Science (SCIE) 등재 - SCOPUS 조회 - Impact Factor (JCR)

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor.

학술저널

issue: Gene therapy [Gene Ther] 2023 Apr; Vol. 30 (3-4), pp. 245-254. Date of Electronic Publication: 2021 Jan 17.
Author: Barbon E; Généthon, 91000, Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare research unit UMR_S951, 91000, Evry, France.
Kawecki C; Laboratory of Hemostasis, Inflammation and Thrombosis, Institut National de la Santé et de la Recherche Médicale UMR_1176, Université Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Marmier S; Généthon, 91000, Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare research unit UMR_S951, 91000, Evry, France.
Sakkal A; Généthon, 91000, Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare research unit UMR_S951, 91000, Evry, France.
Collaud F; Généthon, 91000, Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare research unit UMR_S951, 91000, Evry, France.
Charles S; Généthon, 91000, Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare research unit UMR_S951, 91000, Evry, France.
Ronzitti G; Généthon, 91000, Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare research unit UMR_S951, 91000, Evry, France.
Casari C; Laboratory of Hemostasis, Inflammation and Thrombosis, Institut National de la Santé et de la Recherche Médicale UMR_1176, Université Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Christophe OD; Laboratory of Hemostasis, Inflammation and Thrombosis, Institut National de la Santé et de la Recherche Médicale UMR_1176, Université Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Denis CV; Laboratory of Hemostasis, Inflammation and Thrombosis, Institut National de la Santé et de la Recherche Médicale UMR_1176, Université Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Lenting PJ; Laboratory of Hemostasis, Inflammation and Thrombosis, Institut National de la Santé et de la Recherche Médicale UMR_1176, Université Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Mingozzi F; Généthon, 91000, Evry, France. federico.mingozzi@sparktx.com.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare research unit UMR_S951, 91000, Evry, France. federico.mingozzi@sparktx.com.; Spark Therapeutics, Philadelphia, PA, 19103, USA. federico.mingozzi@sparktx.com.
DB Label: Database : MEDLINE

원문보기: Full Text (Nature) 등재 - Web of Science (SCIE) 등재 - SCOPUS 조회 - Impact Factor (JCR)

Illustrated State-of-the-Art Capsules of the ISTH 2022 Congress.

학술저널

issue: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2022 Jul 06; Vol. 6 (5), pp. e12747. Date of Electronic Publication: 2022 Jul 06 (Print Publication: 2022).
Author: Ariëns RA; University of Leeds Leeds UK.
Hunt BJ; King's Healthcare Partners London UK.
Agbani EO; Department of Physiology and Pharmacology, Cumming School of Medicine University of Calgary Calgary Alberta Canada.
Ahnström J; Centre for Haematology Imperial College London London UK.
Ahrends R; Institute of Analytical Chemistry University of Vienna Vienna Austria.
Alikhan R; Haemostasis & Thrombosis University Hospital of Wales Cardiff UK.
Assinger A; Medical University of Vienna Vienna Austria.
Bagoly Z; Faculty of Medicine, Department of Laboratory Medicine, Division of Clinical Laboratory Sciences and ELKH-DE Neurodegenerative and Cerebrovascular Research Group University of Debrecen Debrecen Hungary.
Balduini A; University of Pavia Pavia Italy.
Barbon E; San Raffaele Telethon Institute for Gene Therapy IRCCS San Raffaele Scientific Institute Milan Italy.
Barrett CD; Division of Acute Care Surgery and Surgical Critical Care, Department of Surgery University of Nebraska Medical Center Omaha Nebraska USA.; Koch Institute, Center for Precision Cancer Medicine Massachusetts Institute of Technology Cambridge Massachusetts USA.; Division of Surgical Critical Care, Department of Surgery, Boston University Medical Center Boston University School of Medicine Boston Massachusetts USA.
Batty P; Royal Free Hospital London UK.
Carneiro JDA; Instituto da Criança do Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo São Paulo Brazil.
Chan WS; University of British Columbia Vancouver British Columbia Canada.
de Maat M; Department of Hematology Erasmus MC Rotterdam The Netherlands.
de Wit K; Queen's University and McMaster University Kingston ON Canada.
Denis C; INSERM UMRS 1176 Le Kremlin-Bicetre Cedex France.
Ellis MH; Hematology Institute and Blood Bank, Meir Medical Center and Sackler School of Medicine Tel Aviv University Tel Aviv Israel.
Eslick R; Haematology Department Canberra Hospital Garran Australian Capital Territory Australia.
Fu H; Division of Hematology, Department of Medicine University of Washington Seattle Washington USA.
Hayward CPM; Hamilton Health Sciences McMaster University Medical Center Hamilton Ontario Canada.
Ho-Tin-Noé B; INSERM Unit 1148 Paris France.
Klok FA; Department of Medicine - Thrombosis and Hemostasis Leiden University Medical Center Leiden The Netherlands.
Kumar R; Dana Farber/Boston Children's Cancer and Blood Disorders Center Boston Massachusetts USA.
Leiderman K; Colorado School of Mines Golden Colorado USA.
Litvinov RI; Department of Cell and Developmental Biology University of Pennsylvania School of Medicine Philadelphia Pennsylvania USA.
Mackman N; UNC Blood Research Center, Division of Hematology, Department of Medicine University of North Carolina at Chapel Hill Chapel Hill North Carolina USA.
McQuilten Z; Monash University Melbourne Victoria Australia.
Neal MD; Trauma and Transfusion Medicine Research Center, Department of Surgery University of Pittsburgh Pittsburgh Pennsylvania USA.
Parker WAE; Cardiovascular Research Unit, Northern General Hospital University of Sheffield Sheffield UK.
Preston RJS; Irish Centre for Vascular Biology, Department of Pharmacy & Biomolecular Sciences Royal College of Surgeons in Ireland Dublin 2 Ireland.
Rayes J; University of Birmingham Birmingham UK.
Rezaie AR; Cardiovascular Biology Research Program Oklahoma Medical Research Foundation Oklahoma City Oklahoma USA.
Roberts LN; King's Thrombosis Centre, Department of Haematological Medicine King's College Hospital NHS Foundation Trust London UK.
Rocca B; Department of Safety and Bioethics, Section of Pharmacology Catholic University School of Medicine Rome Italy.
Shapiro S; Oxford University Hospitals NHS Foundation Trust Oxford UK.; Radcliffe Department of Medicine Oxford University Oxford UK.
Siegal DM; Ottawa Hospital Research Institute and University of Ottawa Ottawa Ontario Canada.
Sousa LP; Departamento de Análises Clínicas e Toxicológicas, Faculdade de Farmácia Universidade Federal de Minas Gerais Belo Horizonte Brazil.
Suzuki-Inoue K; Department of Clinical and Laboratory Medicine University of Yamanashi Yamanashi Japan.
Zafar T; Frontier Medical College Abbotabad Pakistan.; Hemophilia Treatment Center Rawalpindi Pakistan.
Zhou J; Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin China.
DB Label: Database : MEDLINE

원문보기: Full Text (Clinical Key) Open Access (PubMed Central) Full Text (ScienceDirect O/A) 등재 - Web of Science (SCIE) 등재 - SCOPUS 조회 - Impact Factor (JCR)

Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice.

학술저널

issue: Frontiers in genome editing [Front Genome Ed] 2022 Mar 11; Vol. 4, pp. 785698. Date of Electronic Publication: 2022 Mar 11 (Print Publication: 2022).
Author: Lisjak M; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
De Caneva A; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Marais T; Inserm UMRS974, Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Université, Paris, France.
Barbon E; Genethon, Evry, France.; IRCCS San Raffaele Hospital, Milan, Italy.
Biferi MG; Inserm UMRS974, Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Université, Paris, France.
Porro F; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Barzel A; Department of Biochemistry and Molecular Biology, The George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel Aviv, Israel.
Zentilin L; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Kay MA; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA, United States.
Mingozzi F; Genethon, Evry, France.; University Pierre and Marie Curie - Paris 6, INSERM U974, Paris, France.; Spark Therapeutics, Philadelphia, PA, United States.
Muro AF; International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
DB Label: Database : MEDLINE

원문보기: Open Access (PubMed Central)

Visual Acuity, Wavefront Aberrations, and Defocus Curves With an Enhanced Monofocal and a Monofocal Intraocular Lens: A Prospective, Randomized Study.

학술저널

issue: Journal of refractive surgery (Thorofare, N.J. : 1995) [J Refract Surg] 2022 Jan; Vol. 38 (1), pp. 10-20. Date of Electronic Publication: 2022 Jan 01.
Author: Nanavaty MA
Ashena Z
Gallagher S
Borkum S
Frattaroli P
Barbon E
DB Label: Database : MEDLINE