gene-therapy

Documents about gene-therapy

• Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss#831960ClinicalTrials.gov
• Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study#878235ClinicalTrials.gov
• Towards a Comprehensive Screening Approach for Detecting Gene Doping in Racehorses and Sport Horses by Targeting Common and Intrinsic Plasmid Sequences#1039029HAL (France)
• Targeting Neurovascular Pathways: Gene Therapy as a Novel Treatment for Vascular Dementia After Stroke.#150144NCBI PubMed Central
• Gene Therapy in Treating Patients With Stage III or Stage IV Melanoma#154639ClinicalTrials.gov
• Human liver organoids uncover ductular reaction in Wolman disease and inform gene therapy design#307811Europe PMC
• Optimization of AAV tools to target Muller glial cells for retinal gene therapy#314847Europe PMC
• Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness#354015HAL (France)
• Characterization of HIF-1 alpha overexpressing HeLa cells and implications for gene therapy#405350DataCite
• CRISPR/Cas9 Instantaneous Gene Editing Therapy to Intraocular Hypertensive POAG With MYOC Mutation#435824ClinicalTrials.gov
• Phase 1 Trial of Interleukin 12 Gene Therapy for Metastatic Pancreatic Cancer#807697ClinicalTrials.gov
• Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation#890388ClinicalTrials.gov
• Long Term Follow-up Study of AAVAnc80-antiVEGF Gene Therapy#968224ClinicalTrials.gov
• Examining the Impact of Phytoestrogens Supplementation on the Gene Expression, and Premenstrual Syndrome in Primenopausal Women#804779ClinicalTrials.gov
• AAV-based gene therapy with modified HEXB confers lasting therapeutic benefits in GM2 gangliosidosis models.#223008NCBI PubMed Central
• Adebrelimab Plus Chemotherapy as Neoadjuvant Therapy in Resectable NSCLC Harboring Driver Gene Mutations#314339ClinicalTrials.gov
• Targets in Gene Therapy#486512Directory of Open Access Books
• Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002#804611ClinicalTrials.gov
• JS001 Combination Therapy in NSCLC Negative Driving Gene After First-line Chemotherapy.#858196ClinicalTrials.gov
• A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)#862942ClinicalTrials.gov
• Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy#893981ClinicalTrials.gov
• Endocrine Therapy Base on 21-gene RS#968756ClinicalTrials.gov
• Safety and Preliminary Efficacy of NXL-001 in Patients With Ischemic Stroke#981351ClinicalTrials.gov
• A Gene Transfer Study for Hemophilia A#826644ClinicalTrials.gov
• CAR T Cell Receptor Immunotherapy Targeting EGFRvIII for Patients With Malignant Gliomas Expressing EGFRvIII#870470ClinicalTrials.gov
• Long-term Follow-up of Study Participant Treated With Lentiviral-Based Genetically Modified Autologous Cell Product ,AGT103-T#881864ClinicalTrials.gov
• Targeting of Itch by clomipramine or gene therapy improves cognitive defects related to Alzheimer's disease#5586Europe PMC
• Beyond Excitotoxicity: Glutamate-Independent Mechanisms Underlie Noise-Induced Synaptopathy and Compromise Gene Therapy for Hereditary Deafness#231393Europe PMC
• Adeno-Associated Virus Gene Therapy for Spinal Muscular Atrophy Induces Hepatotoxicity via Cytokine and Macrophage Activation.#342608NCBI PubMed Central
• Recombinant adeno-associated virus-mediated HSV-TK/GCV gene therapy for ovarian cancer: an experimental study.#368183NCBI PubMed Central
• Perceptions of Cell and Gene Therapies (CGT) for the Treatment of Cancer#851391ClinicalTrials.gov
• Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug#869220ClinicalTrials.gov
• Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure#880906ClinicalTrials.gov
• Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)#883898ClinicalTrials.gov
• Study Combining Suicide Gene Therapy With Chemoradiotherapy in the Treatment of Non-Metastatic Pancreatic Adenocarcinoma#902535ClinicalTrials.gov
• ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy#908043ClinicalTrials.gov
• Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis#862437ClinicalTrials.gov
• Engineering liposomes via click chemistry for multimodal biomedical applications: Evidence from eukaryotic transfection and antimicrobial light-triggered therapy#424884HAL (France)
• Thailand's Emerging Role in the Cell and Gene Therapy Revolution: A Review of Progress and Potential.#13781NCBI PubMed Central
• IL-12 Gene and in Vivo Electroporation-Mediated Plasmid DNA Vaccine Therapy in Patients With Merkel Cell Cancer#67683ClinicalTrials.gov
• Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy#176853HAL (France)
• 12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65 -mediated inherited retinal dystrophy#308165HAL (France)
• JMJD3 and UTX as key targets for gene-modified mesenchymal stem cell therapy in cartilage tissue engineering#364094HAL (France)
• Replication Data for: Patient preferences in gene therapy development and trial participation for Arrhythmogenic Cardiomyopathy: The UR-HEART Qualitative Study#378235KU Leuven RDR Dataverse OAI Archive
• Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness.#389005NCBI PubMed Central
• EGFR-TKIs Combine Chemotherapy as First-line Therapy for Patients With Advanced EGFR Mutation-positive NSCLC#832710ClinicalTrials.gov
• HEAT Trial (HER2 Antibody Therapy With Lutetium-177)#840309ClinicalTrials.gov
• A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A#842511ClinicalTrials.gov
• Long Term Safety and Efficacy Study of ProSavin in Parkinson's Disease#859776ClinicalTrials.gov
• Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65#894967ClinicalTrials.gov