gene-therapy

Documents about gene-therapy

• Elevated ICAM5 as a promising predictor of poor prognosis in bladder cancer via EMT, immune microenvironment, and therapy resistance#269668PLOS
• Integration of single-cell cis-expression quantitative trait locus and Mendelian randomization analyses identifies a Treg-specific gene for precision therapy in rheumatoid arthritis, gonarthrosis, and gouty arthritis.#320581NCBI PubMed Central
• A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma#424194ClinicalTrials.gov
• Topical gene therapy for severe recessive dystrophic epidermolysis bullosa: clinical outcomes and multidisciplinary management in three patients treated with beremagene geperpavec (B-VEC) in Italy - a case series.#446049NCBI PubMed Central
• Gene-Modified Lymphocytes, High-Dose Aldesleukin, and Vaccine Therapy in Treating Patients With Progressive or Recurrent Metastatic Cancer#623546ClinicalTrials.gov
• Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease#910382ClinicalTrials.gov
• Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59#1007593ClinicalTrials.gov
• Safety and Efficacy of Tipifarnib in Head and Neck Cancer With HRAS Mutations and Impact of HRAS on Response to Therapy#1010033ClinicalTrials.gov
• 3rd Generation GD-2 Chimeric Antigen Receptor and iCaspase Suicide Safety Switch, Neuroblastoma, GRAIN#1045689ClinicalTrials.gov
• C/EBP-β-regulated complement hyperactivation in spleen of SFTSV-infected mice: A clue to targeted complement therapy#13952PLOS
• Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease#845916ClinicalTrials.gov
• A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)#891947ClinicalTrials.gov
• A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)#898193ClinicalTrials.gov
• Phase III Lucanix™ Vaccine Therapy in Advanced Non-small Cell Lung Cancer (NSCLC) Following Front-line Chemotherapy#1010931ClinicalTrials.gov
• Gene and Molecular Pathways of Ozone Treatment Response in Gynecological Tumor Patients With Chronic Pelvic Pain Secondary to Cancer Treatment#871119ClinicalTrials.gov
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)#982731ClinicalTrials.gov
• Phase I Trial of Atezolizumab and Interleukin-12 Gene Therapy in Metastatic Non-Small Cell Lung Cancer With Progression on First-Line Immunotherapy With or Without Chemotherapy#471444ClinicalTrials.gov
• Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis#801380ClinicalTrials.gov
• Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients#873942ClinicalTrials.gov
• A Study Evaluating the Safety of Cal-1 (LVsh5/C46) Drug Product in HIV-1 Infected Patient With High Risk Lymphoma#885227ClinicalTrials.gov
• Characterizing Fatigue Experienced by Cancer Patients Receiving Primary Treatment and Cancer Survivors#910344ClinicalTrials.gov
• Immunoregulatory Effects of Immunoglobulin Induction Therapy in Renal Transplant Recipients#1010193ClinicalTrials.gov
• VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)#1046639ClinicalTrials.gov
• Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy#305462Open Access LMU
• Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17)#825671ClinicalTrials.gov
• BCMA-CD19 cCAR T for the Treatment of Refractory Inflammatory Bowel Disease (IBD)#845504ClinicalTrials.gov
• A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia#857851ClinicalTrials.gov
• Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders#876564ClinicalTrials.gov
• Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss#877117ClinicalTrials.gov
• A novel approach to systemic delivery of therapies in muscular dystrophies through engineering of hematopoietic stem cells#330012HAL (France)
• Predicting BReast cAncer Response to neoadjuVant Therapy by ctDNA Assessment#883791ClinicalTrials.gov
• T Cells Expressing Fully-human Anti-CD19 and Anti-CD20 Chimeric Antigen Receptors for Treating B-cell Malignancies and Hodgkin Lymphoma#875562ClinicalTrials.gov
• Muscle-Specific DNM2 Overexpression Improves Charcot–Marie–Tooth Disease In Vivo and Reveals a Narrow Therapeutic Window in Skeletal Muscle#129366HAL (France)
• Association of µ‐opioid receptor (OPRM1) gene polymorphism with response to naltrexone in alcohol dependence: a systematic review and meta‐analysis#296792Gestión del Repositorio Documental de la Universidad de Salamanca
• A First-in-human Clinical Trial Using a Gene Therapy With Patient's Own Stem Cells to Treat Early Type 1 Diabetes#826307ClinicalTrials.gov
• Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID#860516ClinicalTrials.gov
• Safety and Efficacy of Vesemnogene Lantuparvovec, an AAV-Based Gene Therapy in Young Children Under 24 Months with Spinal Muscular Atrophy in Low- and-Middle- Income Countries. Second Interim Report (August 2025).#314159bioRxiv / medRxiv
• A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1#847146ClinicalTrials.gov
• A Study to Evaluate KIN-3248 in Participants With Advanced Tumors Harboring FGFR2 and//or FGFR3 Gene Alterations#851012ClinicalTrials.gov
• Long-term Effects of Lifestyle Intervention in Obesity and Genetic Influence#882470ClinicalTrials.gov
• Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency#904791ClinicalTrials.gov
• Survival Prolongation by Rationale Innovative Genomics#905060ClinicalTrials.gov
• Charge-switching ionizable lipids lower the toxicity of lipid nanoparticles#922243eScholarship
• From <i>in silico</i> prediction to experimental validation: Identification of drugs and novel synergistic combinations that inhibit growth of inflammatory breast cancer cells#976628PLOS
• Gefitinib With Anlotinib in Advanced Non-squamous NSCLC Patients With Uncleared Plasma ctDNA EGFRm After First-line Treatment With Gefitinib#1046333ClinicalTrials.gov
• A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa#842772ClinicalTrials.gov
• CS-121 APOC3 Base Editing in FCS#868094ClinicalTrials.gov
• Long-term Follow-up of Subjects Exposed to Lentiviral-based CART-EGFRvIII Gene-modified Cellular Therapy Products in Cancer Studies#894669ClinicalTrials.gov
• Synthesis and design of biologically inspired biocompatible iron oxide nanoparticles for biomedical applications#954824Koc University Digital Collections
• An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease#812041ClinicalTrials.gov