gene-therapy

Documents about gene-therapy

• Sickle cells to super cells: the alluring genetic cure for sickle cell disease#277674RUcore, Rutgers University Community Repository
• Human germline enhancement technology and the Zulu ontology on the sanctity of life: an ethico-cultural inquiry.#345727ResearchSpace
• Rescue Reatment of Helicobacter Pylori Based on Fecal Antimicrobial Resistance Gene Testing#349983ClinicalTrials.gov
• Comparative Analysis of the Physicochemical and Biological Characteristics of Freeze-Dried PEGylated Cationic Solid Lipid Nanoparticles#353526Dipòsit Digital de la Universitat de Barcelona
• Defensomes, counter-defensomes, and the remodeling of microbial communities#374590HAL (France)
• ATP8A1 as a Selective Pathologic High-Frequency Target in Temporal Lobe Epilepsy#651656eScholarship
• Long-term Follow-Up Study of RGX-314 and Fellow Eye Substudy#812646ClinicalTrials.gov
• Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease#842474ClinicalTrials.gov
• Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders#870969ClinicalTrials.gov
• Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)#885193ClinicalTrials.gov
• A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])#898318ClinicalTrials.gov
• Mesenchymal Stem/Stromal Cell Therapy in the Treatment of Frailty#982554ClinicalTrials.gov
• Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations#1007841ClinicalTrials.gov
• Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)#1033086ClinicalTrials.gov
• T Cell Receptor Immunotherapy Targeting MAGE-A3 for Patients With Metastatic Cancer Who Are HLA-DP0401 Positive#800372ClinicalTrials.gov
• Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase#906113ClinicalTrials.gov
• Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Mutated or Viral Neoantigens in a Patient With Metastatic Cancer Plus the Administration of Pembrolizumab#968753ClinicalTrials.gov
• Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results#475955HAL (France)
• The Synaptic Clock: SynGAP1 as a Molecular Timer of Postsynaptic Density Consolidation#314720Europe PMC
• Abiraterone Acetate Affects Gene Expression Profile in a Human Male Neuronal Cell Line: Potential Mechanism for Cognitive Deficits with Prostate Cancer Therapy.#409241NCBI PubMed Central
• Aquaporin-1 gene therapy as a novel method to treat radiation-induced salivary gland damage: The case for adeno-associated virus vectors.#458712NCBI PubMed Central
• Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients#828259ClinicalTrials.gov
• Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency#883155ClinicalTrials.gov
• Oral Auranofin for Reduction of Latent Viral Reservoir in Patients With HIV Infection#912958ClinicalTrials.gov
• UCSF Center for Genome Surgery Biobank and Registry#915180ClinicalTrials.gov
• A Long Term Follow-up Study of Subjects Who Received ICM-203 or Matching Placebo#993099ClinicalTrials.gov
• Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"#907951ClinicalTrials.gov
• Common variants of the BRCA1 wild-type allele modify the risk of breast cancer in BRCA1 mutation carriers#179020Lund University Publications
• Polypurine Reverse-Hoogsteen Hairpins as a Tool for Exon Skipping at the Genomic Level in Mammalian Cells#246163Dipòsit Digital de la Universitat de Barcelona
• Immune synapse molecules knockout in induced pluripotent stem cells enables broad NK cell resistance in T cell progeny#308577Kyoto University Research Information Repository
• Immune synapse molecules knockout in induced pluripotent stem cells enables broad NK cell resistance in T cell progeny#308582Kyoto University Research Information Repository
• Cyclophosphamide and Fludarabine Followed by Vaccine Therapy, Gene-Modified White Blood Cell Infusions, and Aldesleukin in Treating Patients With Metastatic Melanoma#426631ClinicalTrials.gov
• Therapeutic Potential of EWSR1-FLI1 Inactivation by CRISPR/Cas9 in Ewing Sarcoma#646694Repisalud
• Targeted Nanoparticle Delivery CRISPR/Cas9: overcoming biological barriers, enhancing stability, and improving therapeutic precision#780679Europe PMC
• Poziotinib in Stage IV Lung Adenocarcinoma With HER2 Mutation (KASTT001)#802082ClinicalTrials.gov
• A Single Dose Clinical Trial to Study the Safety of ART-I02 in Patients With Arthritis#813010ClinicalTrials.gov
• A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome#853436ClinicalTrials.gov
• KCNH2 Polymorphisms on the QTc Interval in Kelantanese Malays Patients Receiving Methadone Maintenance Therapy (MMT)#858490ClinicalTrials.gov
• CD19 CAR T-Cell Therapy for Refractory Systemic Lupus Erythematosus#862379ClinicalTrials.gov
• Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients#888824ClinicalTrials.gov
• AveXis Managed Access Program Cohort for Access to AVXS-101#915775ClinicalTrials.gov
• OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)#994136ClinicalTrials.gov
• The Effect of Non-Surgical Periodontal Therapy on Glycemic Control and Bacterial Levels in a Mexican-American Population With Type 2 Diabetes#904263ClinicalTrials.gov
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)#862999ClinicalTrials.gov
• Growth arrest of <i>Mycobacterium tuberculosis</i> in acidic environments enhances their survival of antibiotic treatment#302049PLOS
• Protection of Genetic Data in Genetic Testing and Gene-Therapy Treatment: IT Law Aspects#462064OpenAlex
• 3RD GENERATION GD2 SPECIFIC CHIMERIC ANTIGEN RECEPTOR TRANSDUCED AUTOLOGOUS NATURAL KILLER T-CELLS FOR NEUROBLASTOMA#800829ClinicalTrials.gov
• Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews#807450ClinicalTrials.gov
• Gene Mutations in Patients With Advanced Prostate Cancer That Is Not Responsive to Hormone Therapy#845609ClinicalTrials.gov
• To Evaluate the Correlation Between the Results of Specific Gene Testing and the Efficacy of Targeted Therapy in Patients With NSCLC#860011ClinicalTrials.gov