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Academic Journal

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.

Subjects: BLOOD coagulation factor IX; ADENO-associated virus; GENE therapy

  • Source: New England Journal of Medicine. 9/26/2024, Vol. 391 Issue 12, p1108-1118. 11p.

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Academic Journal

Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.

Subjects: ADRENOLEUKODYSTROPHY; MAGNETIC resonance imaging; STEM cell transplantation

  • Source: New England Journal of Medicine. 10/10/2024, Vol. 391 Issue 14, p1302-1312. 11p.

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Academic Journal

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.

Subjects: ZINC-finger proteins; SOMATIC mutation; ACUTE myeloid leukemia

  • Source: New England Journal of Medicine. 10/10/2024, Vol. 391 Issue 14, p1287-1301. 15p.

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Academic Journal

In Utero Gene Therapy and its Application in Genetic Hearing Loss.

Subjects: GENE therapy; STEM cell transplantation; OSTEOGENESIS imperfecta

  • Source: Advanced Biology. Oct2024, Vol. 8 Issue 10, p1-14. 14p.

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Academic Journal

Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States: A Cost-Effectiveness Analysis.

Subjects: SICKLE cell anemia; UNITED States. Food & Drug Administration; GENE therapy

  • Source: Annals of Internal Medicine. Feb2024, Vol. 177 Issue 2, p155-164. 11p.

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Academic Journal

Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa.

Subjects: EPIDERMOLYSIS bullosa; GENE therapy; HERPES simplex virus

  • Source: New England Journal of Medicine. 2/8/2024, Vol. 390 Issue 6, p530-535. 6p.

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Academic Journal

PCDH15 dual-AAV gene therapy for deafness and blindness in Usher syndrome type 1F models.

Subjects: USHER'S syndrome; GENE therapy; DEAFNESS

  • Source: Journal of Clinical Investigation. 12/2/2024, Vol. 134 Issue 23, p1-16. 16p.

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Academic Journal

Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.

Subjects: GENOME editing; HEMATOPOIETIC stem cells; GENE expression

  • Source: New England Journal of Medicine. 10/10/2024, Vol. 391 Issue 14, p1358-1359. 2p.

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Academic Journal

Transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: A simulation study for individuals with severe haemophilia A.

Subjects: EMICIZUMAB; GENE therapy; CLINICAL trials

  • Source: Haemophilia. Jul2024, Vol. 30 Issue 4, p905-913. 9p.

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Academic Journal

Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.

Subjects: HEMATOPOIETIC stem cells; CLINICAL trials; ERYTHROCYTES

  • Source: Lancet. 2024, Vol. 404 Issue 10468, p2175-2186. 12p.

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  • 1-10 of  8,516 results for ""GENE therapy""