adeno-associated-virus

Documents about adeno-associated-virus

• Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome#1362221ClinicalTrials.gov
• Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity#176852HAL (France)
• Adeno-Associated Virus-Mediated Gene Delivery for the Treatment of Amyotrophic Lateral Sclerosis and other Motor Neuron Disorders#1100168DataCite
• In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy#1146283HAL (France)
• High-Throughput In Vitro , Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction#1146304HAL (France)
• Gene transfer of arginine kinase to skeletal muscle using adeno-associated virus#1219290HAL (France)
• Gene Therapy for Severe Crigler Najjar Syndrome#1296080ClinicalTrials.gov
• Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations#804093ClinicalTrials.gov
• Durable protection against SIV challenge by adeno-associated virus delivery of Env-specific antibodies#216629bioRxiv / medRxiv
• A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)#994886ClinicalTrials.gov
• Hydrogen Peroxide Imaging via HyPer7 Probe in Adeno Associated Virus-Transduced Primary Mouse Hepatocytes.#457666NCBI PubMed Central
• Myocardial Telomere Recapping Study for Dilated Cardiomyopathy#1443811ClinicalTrials.gov
• Small RNA Barcodes for Sensitive Labeling of AAV Transcripts#651809Europe PMC
• Mechanisms of Adeno-Associated Virus Serotype 9 Vector Characterization and Quality Control through Solid-State Nanopores.#170628NCBI PubMed Central
• Applications of adeno-associated virus for 3D single-cell morphometric analysis in iPSC-derived midbrain organoids.#271974bioRxiv / medRxiv
• 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
• Gene Therapy Clinical Study in Adult PKU#994392ClinicalTrials.gov
• Long-Term Follow Up Study of Subjects Previously Administered HMI 102#1087018ClinicalTrials.gov
• Classification and quantification of full vs. empty adeno-associated virus reference capsids via infrared attenuated total reflection spectroscopy.#222952NCBI PubMed Central
• Adeno-Associated Virus (AAV) for tracking neural development and connectivity in Xenopus#1146749ISTA Publications
• Safety and Immunogenicity Study of tgAAC09, an HIV Vaccine in an Adeno-associated Virus (AAV) Capsid#1185849ClinicalTrials.gov
• Adeno-Associated virus-based approaches for mitochondrial diseases: advances and challenges#431849Springer Nature OA
• The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.#1440010ClinicalTrials.gov
• Safety Study of Subthalamic Nucleus Gene Therapy for Parkinson's Disease#1484666ClinicalTrials.gov
• The interaction between virus-bound KLF4 and host-bound PARP1 directs the localization of wild-type adeno-associated virus type 2 (wtAAV2) to cellular sites of DNA damage.#209523NCBI PubMed Central
• A Study of FVIII Gene Therapy for Hemophilia A#813855ClinicalTrials.gov
• 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
• A Long Term Follow-up Study of Subjects Who Received ICM-203 or Matching Placebo#993099ClinicalTrials.gov
• Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients#873942ClinicalTrials.gov
• Optimizing Cortical Neuron Labeling in vitro for Characterizing Cell Morphology#297895ScholarlyCommons at Penn
• LTFU for Gene Transfer Subjects With Hemophilia B#908832ClinicalTrials.gov
• Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis#912547ClinicalTrials.gov
• Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency#1162047ClinicalTrials.gov
• Identifying adeno‐associated virus ( AAV ) vectors that efficiently target high grade glioma cells, for in vitro monitoring of temporal cell responses#1146274HAL (France)
• A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)#1246691ClinicalTrials.gov
• Enhanced recombinant adeno-associated virus (rAAV) biomanufacturing: Design of experiment (DOE) enabled transfection optimization for maximum full capsid yield and robust scale-up#359175ULRR
• A Study Evaluating the Safety, Tolerability, and Activity of ICM-203 in Subjects With Knee Osteoarthritis.#873077ClinicalTrials.gov
• 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
• Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality#1146309HAL (France)
• Bio-inspired polyphenol-polyelectrolyte WRAP system for geometrically controlled AAV delivery in diverse anatomical sites#425862S-Space: Seoul National University
• A murine model of amyloid‐enhanced tauopathy: Comparison of wild‐type and P301L‐tau#1235116Europe PMC
• A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.#1101493ClinicalTrials.gov
• Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors#1146284HAL (France)
• A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I#1439059ClinicalTrials.gov
• Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy#966498ClinicalTrials.gov
• Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis#1309130ClinicalTrials.gov
• Probing the Capsid: pH-Driven Gating at the AAV 5-Fold Pore and Its Role in Peptide Ligand Binding#1323956Digital Commons @ Michigan Tech
• A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)#891947ClinicalTrials.gov