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Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)—A Fondazione Policlinico Gemelli IRCCS Experience.

• Authors : Favia, Michele; Tarantino, Domenico; Cerbo, Lidia Di

Subjects: TREATMENT of spinal muscular atrophy; INTRAVENOUS therapy; HEALTH status indicatorsITALY

• Source: Hospital Pharmacy; Feb2024, Vol. 59 Issue 1, p39-46, 8p

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Editorial to the Special Issue "Clinical Immunology in Italy, with Special Emphasis to Primary and Acquired Immunodeficiencies: A Commemorative Issue in Honor of Prof. Fernando Aiuti".

• Authors : Aiuti, Alessandro; D'Amelio, Raffaele; Quinti, Isabella

Subjects: PRIMARY immunodeficiency diseases; CLINICAL immunology; MONOCLONAL antibodiesITALY; ROME

• Source: Biomedicines; Dec2023, Vol. 11 Issue 12, p3191, 16p

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Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study.

• Authors : Castaman, Giancarlo; Carulli, Christian; De Cristofaro, Raimondo

Subjects: GENE therapy; DELPHI method; HEMOPHILIAITALY

• Source: Haemophilia; Mar2023, Vol. 29 Issue 2, p435-444, 10p, 5 Charts

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Knowledge and attitudes toward gene therapy of a cohort of Italian patients with hemophilia.

• Authors : Cutica I; Department of Oncology and Hemato-oncology, University of Milan, Milan, Italy.; Mortarino M

Subjects: Hemophilia A*/Hemophilia A*/Hemophilia A*/diagnosis ; Hemophilia A*/Hemophilia A*/Hemophilia A*/therapy ; Hemophilia A*/Hemophilia A*/Hemophilia A*/complications

• Source: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Apr; Vol. 22 (4), pp. 1001-1008. Date of Electronic Publication: 2023 Dec 22.Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836

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Spectrum of Genetic Variants in the Dystrophin Gene: A Single Centre Retrospective Analysis of 750 Duchenne and Becker Patients from Southern Italy.

• Authors : Viggiano, Emanuela; Picillo, Esther; Passamano, Luigia

Subjects: DYSTROPHIN genes; GENETIC variation; MEDICAL geneticsITALY; CAMPANIA (Italy)

• Source: Genes; Jan2023, Vol. 14 Issue 1, p214, 18p

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The Arrival of Gene Therapy for Patients with Hemophilia A.

• Authors : Castaman, Giancarlo; Di Minno, Giovanni; De Cristofaro, Raimondo

Subjects: GENE therapy; BISPECIFIC antibodies; HEMOPHILIACSITALY

• Source: International Journal of Molecular Sciences; Sep2022, Vol. 23 Issue 18, pN.PAG-N.PAG, 18p

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RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy.

• Authors : Sodi, Andrea; Banfi, Sandro; Testa, Francesco

Subjects: GENE therapy; RETINAL diseases; GENETIC disordersITALY

• Source: Orphanet Journal of Rare Diseases; 6/4/2021, Vol. 16 Issue 1, p1-11, 11p

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University of Bologna Researcher Has Published New Study Findings on Oncolytic Virotherapy (Oncolytic Viruses in the Era of Omics, Computational Technologies, and Modeling: Thesis, Antithesis, and Synthesis).

Subjects: ONCOLYTIC virotherapy; RESEARCH personnel; CANCER treatmentITALYUNIVERSITY of Bologna

• Source: Cancer Gene Therapy Week; 1/1/2024, p65-65, 1p

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Study Results from National Institute of Gastroenterology in the Area of Gene Therapy Published (miR-195-5p as Regulator of g-Catenin and Desmosome Junctions in Colorectal Cancer).

Subjects: COLORECTAL cancer; GENE therapy; CELL polarityITALY

• Source: Cancer Gene Therapy Week; 12/28/2023, p59-59, 1p

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University Hospital of Udine Researcher Reports on Findings in Gene Therapy (Recent Developments in Gene Therapy for Neovascular Age-Related Macular Degeneration: A Review).

Subjects: MACULAR degeneration; GENE therapy; UNIVERSITY hospitalsUDINE (Italy); ITALY

• Source: Gene Therapy Weekly; 12/25/2023, p2014-2014, 1p

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