M
Monir Shababi
Researcher at University of Missouri
Publications - 23
Citations - 1410
Monir Shababi is an academic researcher from University of Missouri. The author has contributed to research in topics: Spinal muscular atrophy & SMN1. The author has an hindex of 16, co-authored 23 publications receiving 1304 citations.
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Cardiac defects contribute to the pathology of Spinal Muscular Atrophy models
TL;DR: Whether cardiac tissue is altered in SMA models and whether this could contribute to SMA pathogenesis is determined and the importance of cardiac development and function in these severe models of SMA is demonstrated.
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Spinal muscular atrophy: a motor neuron disorder or a multi‐organ disease?
TL;DR: Clinical and experimental reports are compiled that demonstrate the association between the loss of SMN and peripheral organ deficiency and malfunction and whether defective peripheral organs are a consequence of neuronal damage/muscle atrophy or a direct result ofSMN loss.
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Spinal muscular atrophy: mechanisms and therapeutic strategies
TL;DR: The natural history of SMA has been altered over the past several decades, primarily through supportive care measures, but an effective treatment does not presently exist and the common genetic etiology and recent progress in pre-clinical models suggest that SMA is well-suited for the development of therapeutic regimens.
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Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice.
Jacqueline J. Glascock,Erkan Y. Osman,Tristan H. Coady,Ferrill F. Rose,Monir Shababi,Christian L. Lorson +5 more
TL;DR: The purpose of this video is to demonstrate two different injection paradigms to deliver therapeutic materials into neonatal mice soon after birth which can introduce different therapeutics into the circulatory system, leading to systemic delivery including the CNS.
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Development of a single vector system that enhances trans-splicing of SMN2 transcripts.
TL;DR: It is shown that reducing the competition between endogenous splices sites enhanced the efficiency of trans-splicing, and the ASO-tsRNA vector significantly elevated SMN levels in primary SMA patient fibroblasts, within the central nervous system of SMA mice and increased SMN-dependent in vitro snRNP assembly.