Results 51 to 60 of about 9,435,635 (153)
Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape
Neural Regeneration ResearchMucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive
Courtney J Rouse +2 more
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JIMD Reports, 2023 Late‐onset Pompe disease (LOPD) is a multisystem disorder with significant myopathy. The standard treatment is enzyme replacement therapy (ERT), a therapy that is lifesaving, yet with limitations.
William B. Hannah +6 more
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ApoE gene therapy: an overview and update [PDF]
, 2005 Atherosclerosis remains the leading cause of death in industrialized societies. Apolipoprotein E (ApoE) is an attractive candidate to treat hypercholesterolemia and coronary heart disease, as it is a circulating protein with pleiotropic ...
Owen, J.S.
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List of cellular proteins identified using GeLC-MS analysis of adeno-associated virus vectors.
, 2014 List of cellular proteins identified using GeLC-MS analysis of adeno-associated virus vectors.
Lingxia Chen (519218) +9 more
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ApoE gene therapy to treat hyperlipidemia and atherosclerosis [PDF]
, 2006 Atherosclerosis is the leading cause of death in industrialized countries and is becoming an increasingly worldwide risk to health. Apolipoprotein E (ApoE) is a blood circulating protein with pleiotropic atheroprotective properties that has emerged as a ...
Owen, JS +3 more
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Adeno-Associated Virus Biology
, 2011 Adeno-associated virus (AAV) was first discovered as a contaminant of adenovirus stocks in the 1960s. The development of recombinant AAV vectors (rAAV) was facilitated by early studies that generated infectious molecular clones, determined the sequence ...
Linden, R Michael +3 more
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Adeno‐associated virus vectors for gene therapy—focusing on melanoma
Interdisciplinary MedicineAdeno‐Associated Virus (AAV) vectors have been found to have great potential in the field of gene therapy due to their unique properties. These nonpathogenic vectors exhibit high tissue specificity, low immunogenicity, and sustained gene expression ...
Xingyue Wang +6 more
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Novel Cytotoxic Vectors Based on Adeno-Associated Virus
Toxins, 2010 Vectors based on adeno-associated virus (AAV) are promising tools for gene therapy. The production of strongly toxic vectors, for example for cancer-directed gene transfer, is often unfeasible due to uncontrolled expression of toxic genes in vector ...
Johannes Kohlschütter +2 more
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, 2019 Rare diseases defined by genetic mutations are classic targets for gene therapy. More recently, researchers expanded the use of gene therapy in non-clinical studies to infectious diseases through the delivery of vectorized antibodies to well-defined ...
C. Angelica Medina-Jaszek (8038475) +14 more
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Advances in adeno-associated virus mediated gene therapy in tumors [PDF]
, 2021 Adeno-associated virus (AAV) is a human parvovirus with defects and no disease.AAV vector, as an ideal carrier for the transduction of target genes, has certain advantages in long-term gene correction and gene therapy by transferring foreign bases into ...
YANG Xiao-juan, LI Zhen-hao, GOU Yuan-feng, HUO Xia-qin, PEI Ya-ping, LI Na, LIU Hui-ling
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