gene-therapy

Documents about gene-therapy

• Enhancing the Brain's Barrier: CD73 Gene Therapy for Stroke Protection in Mice / Maria Stamataki ; Betreuer: Tim Magnus#970281Deutsche Nationalbibliothek
• Gene Therapy Clinical Study in Adult PKU#994392ClinicalTrials.gov
• Investigator Initiated Phase 1 Study of TBI-1301#803168ClinicalTrials.gov
• NUDT15/TPMT Multi-gene Guided 6-MP Dosing in Childhood ALL Maintenance Therapy#853820ClinicalTrials.gov
• Next generation AAV-F capsid gene therapy rescues disease pathology in a model of pyruvate dehydrogenase complex deficiency.#362176NCBI PubMed Central
• Etude de la réponse immunitaire virale préexistante anti-AAV chez l'homme#388044HAL (France)
• Innate Immunocompetent hiPSC-Derived Neurospheroids Capture Early CNS Responses to rAAV#784533Europe PMC
• Evaluating the Safety and Preliminary Efficacy of EXG202 Gene Therapy for Neovascular AMD#811177ClinicalTrials.gov
• Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease#828450ClinicalTrials.gov
• The HOPE Biobank Resource (BMT CTN 2402 HOPE)#835845ClinicalTrials.gov
• Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency#854961ClinicalTrials.gov
• A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190#864266ClinicalTrials.gov
• Gene Expression Following Short Term Exposure to Neoadjuvant Endocrine Therapy in Invasive Breast Cancer#884037ClinicalTrials.gov
• Safety and Efficacy of XT-150 for Treatment of Neuropathic Pain#899799ClinicalTrials.gov
• Preliminary Safety and Efficacy of XT-150 in Facet Joint Osteoarthritis#918810ClinicalTrials.gov
• Study of Gene Modified Immune Cells in Patients With Advanced Melanoma#980856ClinicalTrials.gov
• An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)#891254ClinicalTrials.gov
• Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II#902150ClinicalTrials.gov
• Cellular and Molecular Aspects of Parkinson Treatment: Future Therapeutic Perspectives#935861Repositorio de Universidad Autónoma de Chile.
• Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease#897898ClinicalTrials.gov
• Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I#867567ClinicalTrials.gov
• Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering#922264eScholarship
• Radiation-induced esophagitis and lung injury during esophageal squamous cell cancer therapy is correlated to tumor gene expression phenotype.#152200NCBI PubMed Central
• Retinal Impairments in Mice Lacking Both <i>Nxnl1</i> and <i>Nxnl2</i> Genes#157660HAL (France)
• Identification and Prognostic Value of Key Genes in Hepatocellular Carcinoma#804654ClinicalTrials.gov
• Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy#834914ClinicalTrials.gov
• Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy#892481ClinicalTrials.gov
• A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A#896180ClinicalTrials.gov
• Study of EBT-101 in Aviremic HIV-1 Infected Adults on Stable ART#904810ClinicalTrials.gov
• Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)#912780ClinicalTrials.gov
• Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes.#923681NCBI PubMed Central
• Mutlifunctional platforms for gene and drug delivery for cancer therapy#957695Louisiana Tech Digital Commons
• Safety and Efficacy of CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy to Treat Refractory Viral Keratitis#831794ClinicalTrials.gov
• Gene-Modified White Blood Cells Followed By Interleukin-2 and Vaccine Therapy in Treating Patients With Metastatic Melanoma#160178ClinicalTrials.gov
• Synthetic biology of oncolytic bacteria: Comparative microbial chassis and precise killing strategies#311598Europe PMC
• Supplementary Material for: Gene Therapy for Autism Spectrum Disorder: Preclinical Advances, Translational Barriers, and Ethical Dimensions — A Scoping Review#433202Figshare
• Catecholamine-mediated release of miR-133a-3p from adipocytes regulates the onset of chronic primary pain#676446Europe PMC
• Icotinib as the Adjunctive Treatment After Surgery in Stage I-IIIB Lung Adenocarcinoma Patients With EGFR Gene Mutation#845603ClinicalTrials.gov
• The Recurrence Gene Profiles of Adjuvant Osimertinib Therapy in Resected Non-Small-Cell Lung Cancer#858530ClinicalTrials.gov
• Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)#883449ClinicalTrials.gov
• Enzalutamide With or Without Vaccine Therapy for Advanced Prostate Cancer#885466ClinicalTrials.gov
• CD19-CAR Immunotherapy for Childhood Acute Lymphoblastic Leukemia (ALL)#886291ClinicalTrials.gov
• AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa#905602ClinicalTrials.gov
• Clinical Trial Phase I for Theragene in Combination With Chemotherapy for the Locally Advanced Pancreatic Cancer#910217ClinicalTrials.gov
• rRp450-Phase I Trial in Liver Metastases and Primary Liver Tumors#916165ClinicalTrials.gov
• Gold nanoparticle enhancements in electroporation mediated DNA and RNA therapeutics#957760Louisiana Tech Digital Commons
• CARv3-TEAM-E T Cells in Glioblastoma#1010456ClinicalTrials.gov
• Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)#1031150ClinicalTrials.gov
• Oligo-Modified Poly(β-Amino Ester)-Mediated mRNA Delivery: A Protocol for Gene Editing Applications#1055365Springer Nature OA
• Preliminary Results for the Double-dose Adenovirus-mediated Adjuvant Therapy Improving Outcome of Liver Transplantation in Patients With Advanced Hepatocellular Carcinoma#853776ClinicalTrials.gov