adeno-associated-virus

Documents about adeno-associated-virus

• Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity#176852HAL (France)
• 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
• 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
• Classification and quantification of full vs. empty adeno-associated virus reference capsids via infrared attenuated total reflection spectroscopy.#222952NCBI PubMed Central
• Adeno-Associated virus-based approaches for mitochondrial diseases: advances and challenges#431849Springer Nature OA
• 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
• 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
• Bio-inspired polyphenol-polyelectrolyte WRAP system for geometrically controlled AAV delivery in diverse anatomical sites#425862S-Space: Seoul National University
• Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy#966498ClinicalTrials.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
• Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis#801380ClinicalTrials.gov
• Variability in the circulation of cerebrospinal fluid: causes and clinical implications for intraventricular drug delivery#235107HAL (France)
• Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis#862437ClinicalTrials.gov
• Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)#995492ClinicalTrials.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
• Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy#828609ClinicalTrials.gov
• Supplementary Dataset 2. Process and cost-related dataset for comparative analysis of recombinant adeno-associated virus upstream manufacturing platforms#348039KU Leuven RDR Dataverse OAI Archive
• 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)
• A Gene Transfer Study for Hemophilia A#826644ClinicalTrials.gov
• An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis#970063ClinicalTrials.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