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Gene Therapy in Oncology 

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  1. 01-05-1997 | Report

    Interdisciplinary Forum: From Genetic Diagnosis to Gene Therapy in Oncology

    26 September-1 October, 1996, Madrid, Spain
  2. 01-04-2000 | OriginalPaper

    Gene therapy in urologic oncology

  3. 01-04-2014 | OriginalPaper

    Reporting of health-related quality of life (HRQOL) data in oncology trials: a comparison of the European Organization for Research and Treatment of Cancer Quality of Life (EORTC QLQ-C30) and the Functional Assessment of Cancer Therapy-General (FACT-G)

    The assessment of health-related quality of life (HRQOL) in oncology randomised controlled trials (RCTs) through patient-reported outcome (PRO) instruments has seen a steady increase over the last two decades helped, in part, through the …

  4. Journal

    Bulletin of Experimental Biology and Medicine

    This journal publishes only original papers from the leading research institutions on molecular biology of stem and progenitor cells, stem cells as the basis of gene therapy, molecular language of cell-to-cell communication, cytokines, chemokines, growth and other factors, pilot projects on clinical use of stem and progenitor cells.

  5. Journal

    Molecular and Cellular Pediatrics

    Molecular and Cellular Pediatrics is an interdisciplinary open access journal publishing peer-reviewed articles on innovative research in pediatrics. The focus and the topics of this journal cover research areas relevant to pediatrics including …

  6. Journal

    Nuclear Medicine and Molecular Imaging

    It should also be stated clearly in the text that all persons gave their informed consent prior to their inclusion in the study.

  7. 02-05-2024 | Preimplantation Genetic Diagnostics | Online First

    Stringent criteria needed for germline genome editing of human IVF embryos

    Germline genome editing of IVF embryos is controversial because it is not directly health or lifesaving but is intended to prevent genetic diseases in yet-unborn future offspring. The following criteria are thus proposed for future clinical …

  8. Open Access 29-04-2024 | Gene Therapy in Oncology | Online First

    Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

    Sickle cell disease (SCD) refers to a heterogeneous group of genetic abnormalities affecting hemoglobin, the oxygen-carrying protein in red blood cells (RBCs) [ 1 ]. The sickled RBCs characteristic of SCD are more rigid and adherent and have a …

  9. Open Access 18-03-2024 | Hypercholesterolemia | ReviewPaper

    Gene Editing for the Treatment of Hypercholesterolemia

    Atherosclerotic cardiovascular disease is characterized by the narrowing of the vessel lumen due to the accumulation of cholesterol and inflammatory cells within the arterial wall. The progression of this disease puts human subjects at risk of …

  10. Open Access 27-04-2024 | Amyotrophic Lateral Sclerosis | Online First

    Skeletal muscle dysfunction in amyotrophic lateral sclerosis: a mitochondrial perspective and therapeutic approaches

    Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neuromuscular disease that results in the loss of motor neurons and severe skeletal muscle atrophy. The etiology of ALS is linked to skeletal muscle, which can activate a retrograde …

  11. Open Access 28-03-2024 | Hemophilia | OriginalPaper

    In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia

    Hemophilia is a genetic disorder that is caused by mutations in coagulation factor VIII (hemophilia A) or IX (hemophilia B) genes resulting in blood clotting disorders. Despite advances in therapies, such as recombinant proteins and products with …

  12. Open Access 23-03-2024 | Hemophilia | ReviewPaper

    Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany

    Haemophilia is a rare genetic disease that hinders blood clotting. Haemophilia B (HB) is caused by an insufficient concentration of coagulation factor IX (FIX) in the blood. This lack of FIX leads to internal and external bleeding, which can have …

  13. 19-04-2024 | Stem Cell Transplantation | Online First

    Hematopoietic Stem Cell Transplantation for Storage Disorders: Present Status

    Storage disorders are a group of inborn errors of metabolism caused by the defective activity of lysosomal enzymes or transporters. All of these disorders have multisystem involvement with variable degrees of neurological features. Neurological …

  14. Open Access 17-04-2024 | Gene Therapy in Oncology | Online First

    Orthobiologics in knee osteoarthritis, dream or reality?

    Cartilage restoration or repair, also known as orthobiologic therapy, is indicated after the failure of conservative or supportive treatment. However, there is paucity in evidence supporting the efficacy of orthobiologic therapy. The blood-derived …

  15. Open Access 15-04-2024 | Hemophilia | Online First

    Comparative Effectiveness of Valoctocogene Roxaparvovec and Prophylactic Factor VIII Replacement in Severe Hemophilia A

    Hemophilia A (HA), a rare X-linked recessive bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII) protein [ 1 ], affects approximately 17.1 per 100,000 male individuals worldwide [ 2 ]. Standard of care (SOC) for individuals …

  16. Open Access 01-12-2024 | Alzheimer's Disease | OriginalPaper

    Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer’s disease

    Adiponectin (APN) is an adipokine which predominantly expresses in adipocytes with neuroprotective and anti-inflammatory effects. We have recently indicated that circulatory trimeric APN can enter the brain by crossing the blood–brain barrier …

  17. 02-04-2024 | Targeted Therapy | Online First

    Updates on Facioscapulohumeral Muscular Dystrophy (FSHD)

    Facioscapulohumeral dystrophy (FSHD) is a genetically acquired condition that is characterised by gradually progressive asymmetrical muscle weakness of the face, scapular region, upper limbs (humeral) and distal lower limbs (peroneal) [ 1 ]. It is …

  18. Open Access 01-12-2024 | Gene Therapy in Oncology | ReviewPaper

    Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS

    Gene supplementation and editing for neurodegenerative disorders has emerged in recent years as the understanding of the genetic mechanisms underlying several neurodegenerative disorders increases. The most common medium to deliver genetic …

  19. Open Access 01-12-2024 | Phototherapy | Letter

    Crigler-Najjar syndrome: looking to the future does not make us forget the present

    Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore …

  20. Open Access 01-12-2024 | Gene Therapy in Oncology | OriginalPaper

    Values, challenges, and responses associated with high-priced potential cures: perspectives of diverse stakeholders in South Korea

    Recent launches of potential cures, such as cell and gene therapies, have put these technologies at the forefront of the pharmaceutical policy agenda in South Korea, as in many other countries [ 1 , 2 ]. While these technologies have the potential …

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