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Showing 1 to 10 of 10 for “"AAV gene therapy"”.
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Immune responses to AAV gene therapy in the ocular compartment
Adeno-associated virus (AAV) is a viral vector that can be used to deliver therapeutic genes to diseased cells in the eye. Whilst some consider the eye to be an immuneprivileged organ, recent reports have begun to suggest that injection of AAV may elicit local and systemic immune activation. The …
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Improving the Safety and Efficacy of AAV Gene Therapy for Hemophilia A
Gene therapy for hemophilia A (HA) remains an unrealized therapeutic goal due to outstanding questions regarding safety and long-term efficacy after adeno-associated viral (AAV) vector delivery of the F8 gene to hepatocytes. To address questions of safety, we treated privately owned dogs with a …
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OPTIMIZING GENE THERAPY: THE IMPACT OF SMALL MOLECULE MODULATORS ON ADENO-ASSOCIATED VECTOR TRANSDUCTION AND INTEGRATION
Adeno-associated virus (AAV) is a widely used vehicle for gene delivery but a limitation to AAV gene therapy can be inefficient transgene expression from AAV vectors. One approach to improve AAV transduction has been to combine the vector with small molecule modulators that disrupt cellular …
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Adeno-associated virus type 2 as vector for human gene therapy: Characterization of virus-host interactions
Vectors based on adeno-associated virus type 2 (AAV) offer considerable promise for somatic gene therapy of various diseases (e.g. cystic fibrosis, hemophilia B, cancer). Limitations, however, still exist and require further improvement. The study presented here addresses two major problems that …
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Production of AAV vectors for gene therapy : a cost-effectiveness and risk assessment
Gene therapy is a promising modality for the potential treatment of rare Mendelian diseases. To date a number of high profile proof-of-concept studies within the industry have demonstrated the significant disease-correcting promise of this therapeutic strategy. One of the major hurdles that remains …
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MODULATION OF TRPML1/TFEB PATHWAY FOR THE TREATMENT OF WILSON DISEASE
… the P-type ATPase copper transporting B (ATP7B) gene, responsible for copper supply to cuproproteins and removal of excessive copper by biliary excretion. In ATP7B-knockout cells, copper overload triggers autophagy and lysosomal exocytosis via transcription factor EB (TFEB), stimulating copper …
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Rejuvenation of CNS Progenitors via Partial Reprogramming
… OPCs play a critical role in myelin regeneration (remyelination), but, like all stem cells, their regenerative capacity declines with age. OPCs progressively fail to differentiate into mature oligodendrocytes following a demyelinating insult with age, and this failure plays a key role …
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LIVER FIBROSIS IMPAIRS HEPATOCYTE TRANSDUCTION BY AAV VECTORS
Adeno-associated viral vectors (AAVs) are the most promising tools for liver directed gene therapy. However, integrity of hepatic architecture has been considered pre-requisite for efficient gene delivery and clinical studies have been addressed toward patients with no or negligible hepatic damage …
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Cross-Species Evolution of New AAV Variants
<p>Therapeutic gene transfer and genome editing require effective delivery of genetic cargo to target cells and tissues. Recombinant adeno-associated viral (AAV) vectors are a promising delivery platform, but ongoing clinical trials continue to highlight a relatively narrow therapeutic window. …
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3D KIDNEY ORGANOIDS AS A PLATFORM FOR INVESTIGATING MONOGENIC DISEASES AND GENE THERAPY APPROACHES
… human induced pluripotent stem cells (iPSCs) to generate kidney organoids as a 3D model of rare inherited kidney diseases. Human kidney organoids are an invaluable platform for studying renal pathophysiology, developing cell-based therapies, and testing novel therapeutic approaches. We generated …