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Tatyana V. Taksir

Researcher at Genzyme

Publications -  24
Citations -  1270

Tatyana V. Taksir is an academic researcher from Genzyme. The author has contributed to research in topics: Amyotrophic lateral sclerosis & Acid sphingomyelinase. The author has an hindex of 14, co-authored 23 publications receiving 1159 citations.

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Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease.

TL;DR: This study examines the merits of administering a recombinant adeno-associated viral vector designed to deliver small interfering RNA (siRNA) that targets the degradation of the Htt transcript to lower Htt levels and correct the behavioral, biochemical, and neuropathological deficits shown to be associated with the YAC128 mouse model of HD.
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AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease

TL;DR: The findings show that the ASMKO brain is responsive to ASM replacement and that retrograde transport of AAV2 functions as a platform for widespread gene delivery and reversal of pathology in affected brain.
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AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice

TL;DR: It is shown that adeno-associated virus serotype 4 (AAV4)-mediated expression of insulin-like growth factor-1 (IGF-1) or vascular endothelial growth factor (VEGF)-165 in the cellular components of the ventricular system leads to trophic factor delivery throughout the CNS, delayed motor decline and a significant extension of survival in SOD1(G93A) transgenic mice.
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Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis

TL;DR: Intensive investigation of the major determinants of H+ concentration suggests that acidosis is also due in part to the presence of an unknown anion, which provides insights into the pathogenesis of ALS as well as potential targets for drug development.