gene-therapy

Documents about gene-therapy

• TCR Reserved and Power3 (SPPL3) Gene Knock-out Allogeneic CD19-targeting CAR-T Cell Therapy in r/r B Cell Lymphoma#860953ClinicalTrials.gov
• Trial of E10A in Head and Neck Cancer#877847ClinicalTrials.gov
• Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)#891109ClinicalTrials.gov
• Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up#904108ClinicalTrials.gov
• Safety and Efficacy Study of PUMCH-E101 Injection in Subjects with RDH12 Retinopathy#907402ClinicalTrials.gov
• Cilengitide, Temozolomide, and Radiation Therapy in Treating Patients With Newly Diagnosed Glioblastoma and Methylated Gene Promoter Status#967582ClinicalTrials.gov
• The EMPOwER Study Evaluating Multiparameter Gene Testing as a Predictor of Short Term Endocrine Therapy Response in Hormone Receptor Positive Breast Cancers#1010681ClinicalTrials.gov
• Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy#1053173Europe PMC
• Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes#676008eScholarship
• Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB#981099ClinicalTrials.gov
• Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts#917703ClinicalTrials.gov
• Safety and Efficacy of GYA01 (CART84) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Acute Lymphoblastic T Leukemia Patients (T-ALL).#1044278ClinicalTrials.gov
• Long-term Study of ADVM-022 in Neovascular (Wet) AMD [OPTIC-EXT]#912149ClinicalTrials.gov
• T Cell Inflamed Gene Expression Profiling Score-guided Anti PD-1 Therapy (Tislelizumab Monotherapy) for Refractory Solid Cancer Patients Unexposed to Immunotherapy#154647ClinicalTrials.gov
• Bio-inspired Solid Lipid Nanoparticles for therapeutic siRNA delivery targeting the LRRK2 G2019S mutation in a Parkinson’s disease cell model.#215983ResearchSpace
• Status of Alzheimer and Parkinson Diseases Detections and Treatments#452138HAL (France)
• Pilot Study of Autologous T-cells in Patients With Metastatic Pancreatic Cancer#801008ClinicalTrials.gov
• RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)#853228ClinicalTrials.gov
• A Study Evaluating the Safety, Tolerability, and Activity of ICM-203 in Subjects With Knee Osteoarthritis.#873077ClinicalTrials.gov
• Adenovirus Mediated Suicide Gene Therapy With Radiotherapy in Progressive Astrocytoma.#885199ClinicalTrials.gov
• Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy#913626ClinicalTrials.gov
• Magnetofection of green fluorescent protein encoding DNA-bearing polyethyleneimine-coated superparamagnetic iron oxide nanoparticles to human breast cancer cells#955471Koc University Digital Collections
• Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)#992570ClinicalTrials.gov
• A Clinical Study to Evaluate the Safety, and Tolerability of BBM-P002 in the Treatment of Parkinson's Disease#1045942ClinicalTrials.gov
• A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)#1046335ClinicalTrials.gov
• Study of Intra-articular Delivery of tgAAC94 in Inflammatory Arthritis Subjects#1048067ClinicalTrials.gov
• Combination of Mitomycin and Carboplatin in Neoadjuvant Therapy in BRCA-associated Triple-negative Locally Advanced Breast Cancer#884426ClinicalTrials.gov
• Safety Study of MultiGeneAngio in Patients With Chronic Critical Limb Ischemia#813078ClinicalTrials.gov
• Investigating the Efficacy of Autologous, Adipose-derived Mesenchymal Stem Cell Therapy for the Treatment of Sexual Function Impairment in Women Aged 40-50#833540ClinicalTrials.gov
• Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss#20391DOAJ
• Identification of six new susceptibility loci for invasive epithelial ovarian cancer#202825Lund University Publications
• Bio-inspired polyphenol-polyelectrolyte WRAP system for geometrically controlled AAV delivery in diverse anatomical sites#425862S-Space: Seoul National University
• T Cell Receptor Gene Therapy Targeting KK-LC-1 for Gastric, Breast, Cervical, Lung and Other KK-LC-1 Positive Epithelial Cancers#474750ClinicalTrials.gov
• Validation Study of RA-INF-Dx, a Multigene Molecular Test Used to Predict Non-Response to INfliximab Therapy#810297ClinicalTrials.gov
• Therapy With High Clopidogrel Dose or Prasugrel Standard Dose Reduces the Platelet Reactivity in Patients With Genotype Variation RESET GENE Trial#871347ClinicalTrials.gov
• Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD#884044ClinicalTrials.gov
• Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients#884448ClinicalTrials.gov
• Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)#890985ClinicalTrials.gov
• First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis#899499ClinicalTrials.gov
• A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)#913369ClinicalTrials.gov
• A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa#1045181ClinicalTrials.gov
• A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74#870870ClinicalTrials.gov
• Rare Tumor Focused Platform Study of Innovative Therapies and Technologies (PLATFORM2)#839229ClinicalTrials.gov
• Clonal expansion of CAR-T cells harboring lentivector integration in the PLAAT4 gene following anti-CD19 CAR T-cell therapy in a context of cutaneous T-cell lymphoma#342177HAL (France)
• Precision Oncology: Targeting Genomic Alterations and Cancer Signaling with Integrative Multi-Omics, Deep Learning and Network Biology in Medical Oncology#406809OSF
• A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)#858350ClinicalTrials.gov
• Erlotinib Versus Pemetrexed as Second-Line Therapy in Treating Patients With Advanced Lung Adenocarcinoma#885547ClinicalTrials.gov
• Nitroglycerin Plus Radiotherapy Versus Conventional Radiotherapy in Patients With Lung Cancer.#899847ClinicalTrials.gov
• A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia#918423ClinicalTrials.gov
• Gene-Modified T Cells, Vaccine Therapy, and Nivolumab in Treating Patients With Stage IV or Locally Advanced Solid Tumors Expressing NY-ESO-1#146267ClinicalTrials.gov