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Advanced Targeting Strategies for Murine Retroviral and Adeno-associated Viral Vectors
Targeted gene delivery involves broadening viral tropism to infect previously nonpermissive cells, replacing viral tropism to infect a target cell...
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Nonviral Delivery of Cancer Genetic Vaccines
The potential use of genetic vaccines to address numerous diseases including cancer is promising, but currently unrealized. Here, we review...
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Production and Formulation of Adenovirus Vectors
Adenovirus vectors have attracted considerable interest over the past decade, with ongoing clinical development programs for applications ranging...
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Molecular Conjugates
Molecular conjugates are nanometer-sized entities consisting of synthetic materials (lipids, polycations, targeting agents, and so on) and nucleic...
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Adeno-associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications
Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. AAV vectors have successfully been utilized to promote...
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Lentiviral Vectors
We review the use of lentiviral vectors in current human gene therapy applications that involve genetic modification of nondividing tissues with...
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Plasmid Vaccines and Therapeutics: From Design to Applications
In the late 1980s, Vical and collaborators discovered that the injection into tissues of unformulated plasmid encoding various proteins resulted in...
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The Genetics of Cerebral Microbleeds
Cerebral microbleeds are a radiological marker of small areas of haemorrhage in the brain, seen on magnetic resonance imaging sequences sensitive to...
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Intracerebral Hemorrhage and Cerebral Amyloid Angiopathy
Intracerebral hemorrhage (ICH) is the acute manifestation of chronic cerebral small vessel disease. ICH is classified according to the affected brain...
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Genetics of Small Vessel Disease
Cerebral small vessel disease (CSVD) is highly prevalent in the general population and is overall a leading cause of disability. CSVD manifestations...
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Diagnosis, Investigation, Treatment and Management of Young Stroke in Clinic
It has been estimated that approximately 10% of all strokes occur in young adults. Stroke in young people has a significant long-term socioeconomic...
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Multi-Omics Approaches to Discovering Acute Stroke Injury and Recovery Mechanisms
Millions of people suffer an acute stroke each year, resulting in an enormous global burden of residual disability and mortality. Despite decades of...
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Smith-Kingsmore Syndrome (SKS)
Gene defect: mTOR gene
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Popliteal Pterygium Syndrome
The first scientific reports of this syndrome date back to the latter part of the nineteenth century in cases described by Trelat (1869) and Wolff...
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Berlin Ectodermal Dysplasia Syndrome
Ectodermal dysplasia, Berlin type, is a rare congenital disorder consisting of generalized melanoleukoderma, hypodontia, hypotrichosis, infantilism,...
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Potocki-Lupski Syndrome
Potocki-Lupski syndrome (PTLS) is a rare neurodevelopmental disorder that occurs by heterozygous de novo microduplication in the short arm of...
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Mitochondrial Complex IV Deficiency, Nuclear (MC4DN) Types 1–24
Mitochondrial illnesses are a broad, diverse group of molecular disorders affecting the mitochondrial electron transport chain. Mitochondrial complex...
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Interstitial Lung Disease, Nephrotic Syndrome, and Epidermolysis Bullosa (ILNEB)
ILNEB is an extremely rare disease caused by mutations in the ITGA3 gene, which encodes the integrin α3 subunit of the major adhesion...
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Gonadal Dysgenesis, Dysmorphic Facies, Retinal Dystrophy, and Myopathy (GDRM)
Myo-Ectoderma-Gonadal Dysgenesis (MEGD) Syndrome.