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Gene Therapy 

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  1. Open Access 17-05-2024 | Online First

    Gene Therapy for Neurofibromatosis Type 2-Related Schwannomatosis: Recent Progress, Challenges, and Future Directions

    Neurofibromatosis type 2 (NF2)-related schwannomatosis is a rare autosomal dominant monogenic disorder caused by mutations in the NF2 gene. The hallmarks of NF2-related schwannomatosis are bilateral vestibular schwannomas (VS). The current …

  2. Open Access 01-12-2024 | Solid Tumor | Letter

    Unveiling a novel fusion gene enhances CAR T cell therapy for solid tumors

    The efficacy of Adoptive Cell Transfer Therapy (ACT) in combating hematological tumors has been well-documented, yet its application to solid tumors faces formidable hurdles, chief among them being the suboptimal therapeutic response and the …

  3. 11-05-2024 | Gene Therapy in Oncology | Online First

    Raising New Hope for Controlling Seizures in Focal Cortical Dysplasia with Gene Therapy

  4. Open Access 01-12-2024 | OriginalPaper

    Identification of hub genes and therapeutic siRNAs to develop novel adjunctive therapy for Duchenne muscular dystrophy

    Duchenne muscular dystrophy (DMD) is the most frequent hereditary childhood myopathy. It affects predominantly males with an incidence estimated to be about 1 in 3500–5000 live male births [ 1 ]. DMD is characterized by progressive muscle …

  5. Open Access 01-07-2024 | Vulgar Psoriasis | OriginalPaper

    Plexin B2 tissue expression and related gene polymorphisms in psoriasis and their relation to NB-UVB and Acitretin therapy

    Psoriasis is a chronic, immune-mediated, hyperproliferative skin disease. Etiopathogenesis of psoriasis is not well understood. Plexin B2 was found to have effects on CD100-mediated T-cell morphology and expressed in the immune system. It may play …

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

    A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient

    Haemophilia is caused by mutations in the genes encoding coagulation factor VIII (FVIII) or IX (FIX) [ 1 ]. The current standard of care for people with severe haemophilia is life-long prophylaxis with recombinant or plasma-derived coagulation …

  7. Open Access 01-12-2024 | Anthracycline | OriginalPaper

    Variants in structural cardiac genes in patients with cancer therapy-related cardiac dysfunction after anthracycline chemotherapy: a case control study

    Anthracyclines (AC) are one of the most commonly used chemotherapeutic agents, but they are associated with significant toxicities, with cardiotoxicity being the most concerning. Up to 57% of all patients receiving AC develop subclinical …

  8. 15-03-2024 | Myelodysplastic Syndrome | OriginalPaper

    A case of Bloom syndrome manifesting with therapy-related myelodysplastic syndromes harboring a novel BLM gene variant

    Bloom syndrome (BS) is an autosomal recessive genetic disorder caused by variants in the BLM gene. BS is characterized by distinct facial features, elongated limbs, and various dermatological complications including photosensitivity, poikiloderma …

  9. Open Access 01-12-2024 | Targeted Therapy | OriginalPaper

    Clinical impact of panel gene sequencing on therapy of advanced cancers of the digestive system: a retrospective, single center study

    Cancers of the digestive system represent a major fraction of the global tumor burden [ 1 , 2 ]. Despite their high incidence, the therapeutic options for most advanced and metastatic cancers of the digestive system are still limited. Molecular …

  10. 20-03-2024 | OriginalPaper

    Identification of a novel ADCC-related gene signature for predicting the prognosis and therapy response in lung adenocarcinoma

    Globally, lung cancer maintains its unfortunate position as the leading cause of cancer-related mortalities [ 1 ], with lung adenocarcinoma (LUAD) accounting for the most prevalent histological subtype [ 2 , 3 ]. Despite significant advancements in …

  11. 16-03-2024 | Targeted Therapy | EditorialNotes

    From gene panel testing to new molecularly targeted therapy

  12. 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 …

  13. 09-03-2024 | Rivaroxaban | ReviewPaper

    Toward Genetic Testing of Rivaroxaban? Insights from a Systematic Review on the Role of Genetic Polymorphism in Rivaroxaban Therapy

    The introduction of direct oral anticoagulants (DOACs) represents a major medical breakthrough, of which rivaroxaban is an example [ 1 ]. As the first oral direct inhibitor of factor Xa (FXa), rivaroxaban is approved for the treatment and …

  14. 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 …

  15. 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 …

  16. Open Access 01-12-2024 | Glioblastoma | OriginalPaper

    Identification of genetic modifiers enhancing B7-H3-targeting CAR T cell therapy against glioblastoma through large-scale CRISPRi screening

    Glioblastoma multiforme (GBM) is the most aggressive and common primary brain tumor, characterized by rapid growth, diffuse infiltration, and poor prognosis. With a median survival period of only 12–15 months and a 5-year survival rate of less …

  17. 18-04-2024 | Prostate Cancer | Online First

    Same-day post-therapy imaging with a new generation whole-body digital SPECT/CT in assessing treatment response to [177Lu]Lu-PSMA-617 in metastatic castration-resistant prostate cancer

    PSMA (prostate specific membrane antigen) radioligand therapy (RLT) with Lutetium-177 [ 177 Lu]Lu-PSMA-617 (vipivotide tetraxetan) is effective in prolonging radiographic progression free survival (rPFS) and overall survival (OS) in metastatic …

  18. Open Access 01-12-2024 | Solid Tumor | Erratum

    Correction: Comprehensive review of CRISPR‑based gene editing: mechanisms, challenges, and applications in cancer therapy

  19. Open Access 01-12-2024 | Dementia | OriginalPaper

    The effects of individual music therapy in nursing home residents with dementia to improve general well-being: study protocol of a randomized controlled trial

  20. Open Access 01-12-2024 | Ribavirin | OriginalPaper

    Impact of ITPA gene polymorphism for predicting anemia and treatment outcome in HCV infected patients taking Sofosbuvir Ribavirin therapy

    HCV is an important cause of liver-related morbidities and patient death. Pakistan ranks 2nd among countries with approximately 11 million HCV reported cases with day-by-day increasing burden. Around 6% of the Pakistani population is HCV-infected.

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