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The gene therapy plan : taking control of your genetic destiny with diet and lifestyle.

• Authors : Gaynor, Mitchell L., 1956-2015

Subjects: Genetic disorders -- Diet therapy; Nutrition -- Genetic aspects; Cooking for the sick

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Gene therapy

• Source: Funk & Wagnalls New World Encyclopedia. 2024, 1p.

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Pondering the Promise Of Gene Therapy.

• Authors : Smith, Gerry; Contiliano, Tom

Subjects: *HEALTH facilities; GENE therapy; SPOKESPERSONSBIOMARIN Pharmaceutical Inc. BIOMARIN Pharmaceutical Inc. BIOMARIN Pharmaceutical Inc. BMRN

• Source: Bloomberg Businessweek. 2/26/2024, Issue 4813, p10-12. 3p. 1 Graph, 1 Cartoon or Caricature.

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Extension study of novel topical gene therapy reveals positive results.

• Authors : Wehrwein, Peter

Subjects: GENE therapy; SKIN diseases

• Source: Managed Healthcare Executive. Apr2024, Vol. 34 Issue 4, p40-41. 2p.

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Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene therapy.

• Authors : Itzler R; CSL Behring, King of Prussia, Pennsylvania, USA.; Buckner TW

Subjects: Quality of Life* ; Hemophilia B*/Hemophilia B*/Hemophilia B*/psychology ; Hemophilia B*/Hemophilia B*/Hemophilia B*/therapy

• Source: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2024 May; Vol. 30 (3), pp. 709-719. Date of Electronic Publication: 2024 Mar 10.Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up.

• Authors : Roubertie A; CHU Montpellier, Département de Neuropédiatrie, INM, Univ Montpellier, INSERM U 1298, Montpellier, France.; Opladen T

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Aromatic-L-Amino-Acid Decarboxylases*/Aromatic-L-Amino-Acid Decarboxylases*/Aromatic-L-Amino-Acid Decarboxylases*/deficiency ; Aromatic-L-Amino-Acid Decarboxylases*/Aromatic-L-Amino-Acid Decarboxylases*/Aromatic-L-Amino-Acid Decarboxylases*/genetics

• Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2024 May; Vol. 47 (3), pp. 463-475. Date of Electronic Publication: 2023 Jul 17.Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665

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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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Gene therapy corrects the neurological deficits of mice with sialidosis.

• Authors : Hwu WL; Department of Pediatrics, National Taiwan University Hospital, Taipei, Taiwan, ROC. .; Department of Medical Genetics, National Taiwan University Hospital, Taipei, Taiwan, ROC. .

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Neuraminidase*/Neuraminidase*/Neuraminidase*/genetics ; Neuraminidase*/Neuraminidase*/Neuraminidase*/metabolism Neuraminidase 1 deficiency

• Source: Gene therapy [Gene Ther] 2024 May; Vol. 31 (5-6), pp. 263-272. Date of Electronic Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy.

• Authors : Miesbach W; Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany. Electronic address: .; Konkle B

Subjects: Hemophilia A*/Hemophilia A*/Hemophilia A*/genetics ; Hemophilia A*/Hemophilia A*/Hemophilia A*/therapy ; Hemophilia A*/Hemophilia A*/Hemophilia A*/blood

• Source: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 May; Vol. 22 (5), pp. 1510-1515. Date of Electronic Publication: 2024 Jan 18.Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836

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Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea.

• Authors : Jeong H; Pharmaceutical Licensing, Department of Pharmaceutical Industry, Chung-Ang University, 06974, Seoul, South Korea.; Purja S

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; European Union*; Republic of Korea

• Source: Gene therapy [Gene Ther] 2024 May; Vol. 31 (5-6), pp. 242-254. Date of Electronic Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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