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Academic Journal

[How safe is gene therapy? : Second death after Duchenne therapy].

  • Authors : Horn S; Forschungsabteilung Zell- und Gentherapie, Klinik für Stammzelltransplantation, Zentrum für Onkologie, Universitätsklinikum Hamburg-Eppendorf, 20246, Hamburg, Deutschland.; Fehse B

Subjects: Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/genetics ; Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/Muscular Dystrophy, Duchenne*/therapy ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/adverse effects

  • Source: Innere Medizin (Heidelberg, Germany) [Inn Med (Heidelb)] 2024 Jun; Vol. 65 (6), pp. 617-623. Date of Electronic Publication: 2024 May 15.Publisher: Springer Medizin Country of Publication: Germany NLM ID: 9918384885306676 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Academic Journal

Welcoming the Era of Gene Editing in Medicine.

  • Authors : Daley GQ; From Boston Children's Hospital and Harvard Medical School - both in Boston.

Subjects: Anemia, Sickle Cell*/Anemia, Sickle Cell*/Anemia, Sickle Cell*/genetics ; Anemia, Sickle Cell*/Anemia, Sickle Cell*/Anemia, Sickle Cell*/therapy ; CRISPR-Cas Systems*

  • Source: The New England journal of medicine [N Engl J Med] 2024 May 09; Vol. 390 (18), pp. 1642-1645. Date of Electronic Publication: 2024 Apr 24.Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print-Electronic Cited Medium:

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Academic Journal

Non-clinical safety assessment of novel drug modalities: Genome safety perspectives on viral-, nuclease- and nucleotide-based gene therapies.

  • Authors : Lemmens M; Biomedical Research, Novartis, Basel 4057, Switzerland. Electronic address: .; Dorsheimer L

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/adverse effects ; Gene Editing*/Gene Editing*/Gene Editing*/methods

  • Source: Mutation research. Genetic toxicology and environmental mutagenesis [Mutat Res Genet Toxicol Environ Mutagen] 2024 May-Jun; Vol. 896, pp. 503767. Date ofPublisher: Elsevier Country of Publication: Netherlands NLM ID: 101632149 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Academic Journal

[A systemic gene therapy for the treatment of cystic fibrosis].

  • Authors : Boillot L; Université de Tours, Master 2 infectiologie, immunité, vaccinologie et biomédicaments, Tours, France.; Costes F

Subjects: Cystic Fibrosis*/Cystic Fibrosis*/Cystic Fibrosis*/therapy ; Cystic Fibrosis*/Cystic Fibrosis*/Cystic Fibrosis*/genetics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods

  • Source: Medecine sciences : M/S [Med Sci (Paris)] 2024 May; Vol. 40 (5), pp. 467-470. Date of Electronic Publication: 2024 May 31.Publisher: EDK Country of Publication: France NLM ID: 8710980 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1958-5381

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Academic Journal

Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results.

  • Authors : Sevigny J; Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, New York, NY, USA. .; Uspenskaya O

Subjects: Frontotemporal Dementia*/Frontotemporal Dementia*/Frontotemporal Dementia*/genetics ; Frontotemporal Dementia*/Frontotemporal Dementia*/Frontotemporal Dementia*/therapy ; Frontotemporal Dementia*/Frontotemporal Dementia*/Frontotemporal Dementia*/cerebrospinal fluid

  • Source: Nature medicine [Nat Med] 2024 May; Vol. 30 (5), pp. 1406-1415. Date of Electronic Publication: 2024 May 14.Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet

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Academic Journal

Highly Bioadaptable Hybrid Conduits with Spatially Bidirectional Structure for Precision Nerve Fiber Regeneration via Gene Therapy.

  • Authors : Zhao R; Orthopedics Research Institute, Department of Orthopedics, West China Hospital, Sichuan University, Chengdu, 610041, P. R. China.; Trauma Medical Center, Department of Orthopedics Surgery, West China Hospital, Sichuan University, Chengdu, 610041, China.

Subjects: Nerve Regeneration*/Nerve Regeneration*/Nerve Regeneration*/physiology ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods; Animals

  • Source: Advanced science (Weinheim, Baden-Wurttemberg, Germany) [Adv Sci (Weinh)] 2024 May; Vol. 11 (19), pp. e2309306. Date of Electronic Publication: 2024 Mar 14.Publisher: WILEY-VCH Country of Publication: Germany NLM ID: 101664569 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2198-3844

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Academic Journal

Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial.

Subjects: Angina Pectoris*/Angina Pectoris*/Angina Pectoris*/therapy ; Angina Pectoris*/Angina Pectoris*/Angina Pectoris*/physiopathology ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/adverse effects

  • Source: Circulation. Cardiovascular interventions [Circ Cardiovasc Interv] 2024 May; Vol. 17 (5), pp. e014054. Date of Electronic Publication: 2024 May 02.Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 101499602 Publication Model: Print-Electronic Cited Medium:

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Academic Journal

Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa.

  • Authors : Munung NS; Division of Human Genetics, Faculty of Health Sciences, University of Cape Town, Cape Town, South Africa. .; Nnodu OE

Subjects: Anemia, Sickle Cell*/Anemia, Sickle Cell*/Anemia, Sickle Cell*/therapy ; Anemia, Sickle Cell*/Anemia, Sickle Cell*/Anemia, Sickle Cell*/genetics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/ethics

  • Source: Gene therapy [Gene Ther] 2024 May; Vol. 31 (5-6), pp. 202-208. Date of Electronic Publication: 2023 Nov 27.Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Academic Journal

Remuneration of donors for cell and gene therapies: an update on the principles and perspective of the World Marrow Donor Association.

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/economics ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Tissue Donors*

  • Source: Bone marrow transplantation [Bone Marrow Transplant] 2024 May; Vol. 59 (5), pp. 580-586. Date of Electronic Publication: 2024 Feb 23.Publisher: Nature Publishing Group Country of Publication: England NLM ID: 8702459 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Academic Journal

Which patients should be considered for gene therapy.

  • Authors : Miesbach W; Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany.

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/adverse effects ; Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Hemophilia A*/Hemophilia A*/Hemophilia A*/therapy

  • Source: Journal of viral hepatitis [J Viral Hepat] 2024 Apr; Vol. 31 Suppl 1, pp. 9-13.Publisher: Blackwell Scientific Publications Country of Publication: England NLM ID: 9435672 Publication Model: Print Cited Medium: Internet ISSN:

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  • 1-10 of  54,734 results for ""Genetic Therapy""