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Therapy: Gene Therapy
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Total 388 results found since Jan 2013.
Peripherally delivered Adeno-associated viral vectors for spinal cord injury repair
In conclusion, in the future, minimally invasive administration of AAVs may improve recovery after SCI with minimal side effects.PMID:34896114 | DOI:10.1016/j.expneurol.2021.113945
Source: Experimental Neurology - December 13, 2021 Category: Neurology Authors: Jared D Sydney-Smith Aline B Spejo Philippa M Warren Lawrence D F Moon Source Type: research
Clinical features, pathogenesis, and management of stroke-like episodes due to MELAS
AbstractMitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes (MELAS) is a disease that should be considered as a differential diagnosis to acute ischemic stroke taking into account its onset pattern and neurological symptoms, which are similar to those of an ischemic stroke. Technological advancements in neuroimaging modalities have greatly facilitated differential diagnosis between stroke and MELAS on diagnostic imaging. Stroke-like episodes in MELAS have the following features: (1) symptoms are neurolocalized according to lesion site; (2) epileptic seizures are often present; (3) lesion distri...
Source: Metabolic Brain Disease - November 15, 2021 Category: Neurology Source Type: research
Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases
This article mainly focuses on the delivering modes of genetic materials in the CNS, which includes viral and non-viral vectors and their application in gene therapy. Despite the many clinical trials conducted so far, data have shown disappointing outcomes. The efforts done to improve outcomes, efficacy, and safety in the identification of targets in various neurological disorders are also discussed here. Adapting gene therapy as a new therapeutic approach for treating neurological disorders seems to be promising, with early detection and delivery of therapy before the neuron is lost, helping a lot the development of new t...
Source: Molecular Medicine - October 16, 2021 Category: Molecular Biology Authors: Della Grace Thomas Parambi Khalid Saad Alharbi Rajesh Kumar Seetha Harilal Gaber El-Saber Batiha Nat ália Cruz-Martins Omnia Magdy Arafa Musa Dibya Sundar Panda Bijo Mathew Source Type: research
Fabry disease - current data and therapeutic approaches
Rom J Morphol Embryol. 2021 Jan-Mar;62(1):5-11. doi: 10.47162/RJME.62.1.01.ABSTRACTFabry disease represents an X-linked inherited disorder resulting in the accumulation of globotriaosylceramide (Gb3). This review explains the clinical manifestations and the possible therapies for this condition. Fabry disease is considered the second most frequent lysosomal storage disease. More than 1000 mutations of the galactosidase alpha (GLA) gene associated with this disorder have been identified. Pain, either episodic crises or chronic pain, is one of the earliest symptoms in Fabry disease. Gastrointestinal, ocular, ear or skeletal ...
Source: Romanian Journal of Morphology and Embryology - October 5, 2021 Category: General Medicine Authors: Ilie Robert Dinu Ştefan George Firu Source Type: research
