Results 31 to 40 of about 144,727 (384)
Frontiers in Molecular Neuroscience, 2016 Leukodystrophies are hereditary central white matter disorders caused by oligodendrocyte dysfunction. Recent clinical trials for some of these devastating neurological conditions have employed an ex vivo gene therapy approach that showed improved ...
Georg evon Jonquieres +8 more
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AAV Vectors for the Nucleolus [PDF]
Molecular Therapy, 2012 During the replication of wild-type adeno-associated virus (AAV), viral capsids are assembled in the nucleolus,1 and the capsid proteins interact with the nucleolar proteins nucleolin (NCL) and nucleophosmin (NPM1), possibly promoting their transport from the cytoplasm into the nucleolus.2,3 In the case of replication-incompetent AAV vectors, the ...
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Frontiers in Microbiology, 2011 Adeno-associated viral (AAV) vector mediated gene replacement for the treatment of muscular dystrophy represents a promising therapeutic strategy in modern medicine.
Zejing eWang +6 more
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Perinatal Gene Transfer to the Liver [PDF]
, 2011 The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM +6 more
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Nature Biotechnology, 2020 Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine factor VIII (AAV-cFVIII) corrected the FVIII deficiency to 1.9–11.3% of ...
Giang N. Nguyen +14 more
semanticscholar +1 more source
Frontiers in Molecular Biosciences, 2023 Huntington’s disease (HD) is a multi-tissue failure disorder for which there is no cure. We have previously shown an effective therapeutic approach limited mainly to the central nervous system, based on a synthetic zinc finger (ZF) transcription ...
Michal Mielcarek +3 more
doaj +1 more source
Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9 [PDF]
, 2018 Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle wasting disease caused by mutations in the DMD gene. In 51% of DMD cases, a reading frame is disrupted because of deletion of several exons.
Cappellari, O +9 more
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Seek and destroy: targeted adeno-associated viruses for gene delivery to hepatocellular carcinoma
Drug Delivery, 2017 Hepatocellular carcinoma (HCC) is the most common form of primary liver cancer with high incidence globally. Increasing mortality and morbidity rates combined with limited treatment options available for advanced HCC press for novel and effective ...
Bijay Dhungel +3 more
doaj +1 more source
Dysfunction of cortical GABAergic neurons leads to sensory hyper-reactivity in a Shank3 mouse model of ASD. [PDF]
, 2020 Hyper-reactivity to sensory input is a common and debilitating symptom in individuals with autism spectrum disorders (ASD), but the neural basis underlying sensory abnormality is not completely understood.
Chen, Naiyan +16 more
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Annual Meeting of the Berkeley Linguistics Society, 1995 Proceedings of the Twenty-First Annual Meeting of the Berkeley Linguistics Society: General Session and Parasession on Historical Issues in Sociolinguistics/Social Issues in Historical Linguistics (1995)
Patricia Cukor-Avila, Guy Bailey
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